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Neuropatia sensitiva e autonômica hereditária
ORPHA:140471CID-11 · 8C21DOENÇA RARA
Esta doença tem causa genética. Recomendamos procurar um médico geneticista para diagnóstico, exames genéticos e aconselhamento familiar.
Sistema ósseo
Também conhecida comoHSAN

Um caso de neuropatia periférica sensorial causada por uma modificação hereditária do genoma do indivíduo.

Última verificação: Fonte: sem afirmação sobre o SUS a verificarEm construçãohistóricoSugerir correção
Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

💬
EM LINGUAGEM SIMPLES

A insensibilidade congênita à dor com anidrose (CIPA) tem duas características: a incapacidade de sentir dor e temperatura, e a diminuição ou ausência de suor (anhidrose). Esta condição também é conhecida como neuropatia sensorial e autonômica hereditária tipo IV. Os sinais e sintomas da CIPA aparecem cedo, geralmente ao nascer ou durante a infância, mas com atenção médica cuidadosa, as pessoas com a condição podem viver até a idade adulta.

A incapacidade de sentir dor e temperatura frequentemente leva a lesões graves repetidas. Lesões auto‑infligidas não intencionais são comuns em pessoas com CIPA, tipicamente ao morder a língua, os lábios ou os dedos, o que pode levar à amputação espontânea da área afetada. Além disso, as pessoas com CIPA cicatrizam lentamente lesões de pele e osso. Trauma repetido pode levar a infecções ósseas crônicas (osteomielite) ou a uma condição chamada articulações de Charcot, na qual os ossos e os tecidos ao redor das articulações são destruídos.

Normalmente, o suor ajuda a resfriar a temperatura corporal. Contudo, em pessoas com CIPA, a anidrose frequentemente causa febres recorrentes extremamente altas (hiperpirexia) e convulsões provocadas por alta temperatura (convulsões febris).

Além das características principais, há outros sinais e sintomas da CIPA. Muitas pessoas com a condição apresentam pele espessa e coriácea (liquenificação) nas palmas das mãos ou unhas dos dedos das mãos ou dos pés deformadas. Elas também podem ter áreas no couro cabeludo onde o cabelo não cresce (hipotricose). Cerca de metade das pessoas com CIPA apresentam sinais de hiperatividade ou instabilidade emocional, e muitas pessoas com a condição têm deficiência intelectual. Algumas pessoas com CIPA têm tônus muscular fraco (hipotonia) quando são jovens, mas a força e o tônus muscular tornam‑se mais normais à medida que envelhecem.

Fonte: MedlinePlus Genetics (NLM/NIH), traduzido
📋
Informacoes curadas por IA — podem conter imprecisoes

Um caso de neuropatia periférica sensorial causada por uma modificação hereditária do genoma do indivíduo.

Pesquisas ativas
42 ensaios
170 total registrados no ClinicalTrials.gov
Publicações científicas
421 artigos
Último publicado: 2026 Jan 1
🏥
SUS: Sem cobertura SUSScore: 0%
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Entender a doença

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Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🦴
Ossos e articulações
42 sintomas
🧠
Neurológico
25 sintomas
💪
Músculos
19 sintomas
😀
Face
13 sintomas
👁️
Olhos
10 sintomas
🧬
Pele e cabelo
8 sintomas

+ 118 sintomas em outras categorias

Características mais comuns

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Febre recorrente
—
Olho profundamente inserido
—
Achatamento malar
—
Anormalidade da gengiva
—
Anormalidade da dentição
—
Anormalidade da morfologia da epífise
279sintomas
Sem dados (279)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 279 características clínicas mais associadas, ordenadas por frequência.

Febre recorrenteRecurrent fever
Achatamento malarMalar flattening
Anormalidade da gengivaAbnormality of the gingiva
Anormalidade da dentiçãoAbnormality of the dentition

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico421PubMed
Últimos 10 anos200publicações
Pico202228 papers
Linha do tempo
2026Hoje · 2026🧪 1994Primeiro ensaio clínico📈 2022Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

18 genes identificados com associação a esta condição.

Curadoria gene-doença

fontes oficiais
RETREG1 PRDM12NGF
RETREG1NGF
SCN11ASodium channel protein type 11 subunit alphaDisease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (2)
Interaction between L1 and AnkyrinsPhase 0 - rapid depolarisation
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory and autonomic, 7

A form of hereditary sensory and autonomic neuropathy, a genetically and clinically heterogeneous group of disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and by sensory and/or autonomic abnormalities. HSAN7 is characterized by congenital inability to experience pain resulting in self-mutilations, slow-healing wounds, and multiple painless fractures. mild muscle weakness, delayed motor development, slightly reduced motor and sensory nerve conduction velocities, hyperhidrosis and gastrointestinal dysfunction.

EXPRESSÃO TECIDUAL(Baixa expressão)
Baço
3.3 TPM
Testículo
3.0 TPM
Cólon sigmoide
1.1 TPM
Nervo tibial
0.8 TPM
Tecido adiposo
0.7 TPM
OUTRAS DOENÇAS (6)
hereditary sensory and autonomic neuropathy type 7familial episodic pain syndrome with predominantly lower limb involvementparoxysmal extreme pain disorderchannelopathy-associated congenital insensitivity to pain, autosomal recessive
HGNC:10583UniProt:Q9UI33
SCN9ASodium channel protein type 9 subunit alphaCandidate gene tested inTolerante
VIAS BIOLÓGICAS (3)
Interaction between L1 and AnkyrinsPhase 0 - rapid depolarisationSensory perception of sweet, bitter, and umami (glutamate) taste
MECANISMO DE DOENÇA

Primary erythermalgia

Autosomal dominant disease characterized by recurrent episodes of severe pain associated with redness and warmth in the feet or hands.

EXPRESSÃO TECIDUAL(Tecido-específico)
Testículo
8.9 TPM
Nervo tibial
8.9 TPM
Hipotálamo
7.5 TPM
Cólon sigmoide
6.3 TPM
Baço
5.3 TPM
OUTRAS DOENÇAS (7)
channelopathy-associated congenital insensitivity to pain, autosomal recessiveparoxysmal extreme pain disorderprimary erythermalgiaobsolete sodium channelopathy-related small fiber neuropathy
HGNC:10597UniProt:Q15858
DNMT1DNA (cytosine-5)-methyltransferase 1Disease-causing germline mutation(s) inAltamente restrito
VIAS BIOLÓGICAS (7)
STAT3 nuclear events downstream of ALK signalingNuclear events stimulated by ALK signaling in cancerDefective pyroptosisPRC2 methylates histones and DNADNA methylation
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory, 1E

A neurodegenerative disorder characterized by adult onset of progressive peripheral sensory loss associated with progressive hearing impairment and early-onset dementia.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
84.0 TPM
Testículo
56.6 TPM
Cérebro - Hemisfério cerebelar
54.8 TPM
Cerebelo
53.6 TPM
Fibroblastos
46.5 TPM
OUTRAS DOENÇAS (2)
autosomal dominant cerebellar ataxia, deafness and narcolepsyhereditary sensory neuropathy-deafness-dementia syndrome
HGNC:2976UniProt:P26358
ATL1Atlastin-1Disease-causing germline mutation(s) inRestrito
MECANISMO DE DOENÇA

Spastic paraplegia 3, autosomal dominant

A form of spastic paraplegia, a neurodegenerative disorder characterized by a slow, gradual, progressive weakness and spasticity of the lower limbs. Rate of progression and the severity of symptoms are quite variable. Initial symptoms may include difficulty with balance, weakness and stiffness in the legs, muscle spasms, and dragging the toes when walking. In some forms of the disorder, bladder symptoms (such as incontinence) may appear, or the weakness and stiffness may spread to other parts of the body.

OUTRAS DOENÇAS (3)
hereditary spastic paraplegia 3Aneuropathy, hereditary sensory, type 1Dhereditary sensory and autonomic neuropathy type 1
HGNC:11231UniProt:Q8WXF7
CLCF1Cardiotrophin-like cytokine factor 1Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (1)
IL-6-type cytokine receptor ligand interactions
MECANISMO DE DOENÇA

Crisponi/Cold-induced sweating syndrome 2

An autosomal recessive disorder characterized by profuse sweating induced by cool surroundings (temperatures of 7 to 18 degrees Celsius). Patients manifest, in the neonatal period, orofacial weakness with impaired sucking and swallowing, resulting in poor feeding. Affected infants show a tendency to startle, with contractions of the facial muscles in response to tactile stimuli or during crying, trismus, abundant salivation, and opisthotonus. These features are referred to as Crisponi syndrome and can result in early death in infancy. Patients who survive into childhood have hyperhidrosis, mainly of the upper body, in response to cold temperatures, and sweat very little with heat. Additional abnormalities include a high-arched palate, nasal voice, depressed nasal bridge, inability to fully extend the elbows and kyphoscoliosis.

OUTRAS DOENÇAS (3)
cold-induced sweating syndrome 2cold-induced sweating syndromeCold-induced sweating syndrome 1
HGNC:17412UniProt:Q9UBD9
CRLF1Cytokine receptor-like factor 1Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (1)
Interleukin-27 signaling
MECANISMO DE DOENÇA

Crisponi/Cold-induced sweating syndrome 1

An autosomal recessive disorder characterized by profuse sweating induced by cool surroundings (temperatures of 7 to 18 degrees Celsius). Patients manifest, in the neonatal period, orofacial weakness with impaired sucking and swallowing, resulting in poor feeding. Affected infants show a tendency to startle, with contractions of the facial muscles in response to tactile stimuli or during crying, trismus, abundant salivation, and opisthotonus. These features are referred to as Crisponi syndrome and can result in early death in infancy. Patients who survive into childhood have hyperhidrosis, mainly of the upper body, in response to cold temperatures, and sweat very little with heat. Additional abnormalities include a high-arched palate, nasal voice, depressed nasal bridge, inability to fully extend the elbows and kyphoscoliosis.

EXPRESSÃO TECIDUAL(Ubíquo)
Artéria tibial
361.1 TPM
Artéria coronária
165.9 TPM
Aorta
121.4 TPM
Tireoide
63.7 TPM
Nervo tibial
45.4 TPM
OUTRAS DOENÇAS (3)
Cold-induced sweating syndrome 1idiopathic achalasiacold-induced sweating syndrome
HGNC:2364UniProt:O75462
WNK1Serine/threonine-protein kinase WNK1Disease-causing germline mutation(s) inAltamente restrito
VIAS BIOLÓGICAS (1)
Stimuli-sensing channels
MECANISMO DE DOENÇA

Pseudohypoaldosteronism 2C

An autosomal dominant disorder characterized by severe hypertension, hyperkalemia, hyperchloremia, mild hyperchloremic metabolic acidosis in some cases, and correction of physiologic abnormalities by thiazide diuretics.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Ubíquo)
Brain Spinal cord cervical c-1
133.1 TPM
Linfócitos
83.2 TPM
Skin Sun Exposed Lower leg
76.8 TPM
Substância negra
68.2 TPM
Vagina
65.2 TPM
OUTRAS DOENÇAS (3)
neuropathy, hereditary sensory and autonomic, type 2Apseudohypoaldosteronism type 2Chereditary sensory and autonomic neuropathy type 2
HGNC:14540UniProt:Q9H4A3
SPTLC1Serine palmitoyltransferase 1Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (1)
Sphingolipid de novo biosynthesis
MECANISMO DE DOENÇA

Amyotrophic lateral sclerosis 27, juvenile

A form of amyotrophic lateral sclerosis, a neurodegenerative disorder affecting upper motor neurons in the brain and lower motor neurons in the brain stem and spinal cord, resulting in fatal paralysis. Sensory abnormalities are absent. The pathologic hallmarks of the disease include pallor of the corticospinal tract due to loss of motor neurons, presence of ubiquitin-positive inclusions within surviving motor neurons, and deposition of pathologic aggregates. The etiology of amyotrophic lateral sclerosis is likely to be multifactorial, involving both genetic and environmental factors. The disease is inherited in 5-10% of the cases. ALS27 is an autosomal dominant form manifesting as toe walking and gait abnormalities in early childhood.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
55.6 TPM
Esôfago - Mucosa
37.6 TPM
Cervix Ectocervix
36.9 TPM
Ovário
36.8 TPM
Vagina
36.5 TPM
OUTRAS DOENÇAS (4)
neuropathy, hereditary sensory and autonomic, type 1Aamyotrophic lateral sclerosis 27, juvenilejuvenile amyotrophic lateral sclerosishereditary sensory and autonomic neuropathy type 1
HGNC:11277UniProt:O15269
PRDM12PR domain zinc finger protein 12Disease-causing germline mutation(s) inTolerante
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory and autonomic, 8

A form of hereditary sensory and autonomic neuropathy, a genetically and clinically heterogeneous group of disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and by sensory and/or autonomic abnormalities. HSAN8 patients manifest congenital insensitivity to pain resulting in ulceration to the fingers, tongue, lips, and other distal appendages. Some patients may also have decreased sweating and tear production.

EXPRESSÃO TECIDUAL(Baixa expressão)
Hipotálamo
0.8 TPM
Cérebro - Hemisfério cerebelar
0.7 TPM
Cerebelo
0.6 TPM
Brain Frontal Cortex BA9
0.6 TPM
Córtex cerebral
0.6 TPM
OUTRAS DOENÇAS (1)
congenital insensitivity to pain-hypohidrosis syndrome
HGNC:13997UniProt:Q9H4Q4
DSTDystoninDisease-causing germline mutation(s) inAltamente restrito
VIAS BIOLÓGICAS (6)
Type I hemidesmosome assemblyRND1 GTPase cycleRHOU GTPase cycleRHOV GTPase cycleRND3 GTPase cycle
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory and autonomic, 6

A form of hereditary sensory and autonomic neuropathy, a genetically and clinically heterogeneous group of disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and by sensory and/or autonomic abnormalities. HSAN6 is a severe autosomal recessive disorder characterized by neonatal hypotonia, respiratory and feeding difficulties, lack of psychomotor development, and autonomic abnormalities including labile cardiovascular function, lack of corneal reflexes leading to corneal scarring, areflexia, and absent axonal flare response after intradermal histamine injection.

EXPRESSÃO TECIDUAL(Ubíquo)
Cervix Ectocervix
76.7 TPM
Skin Not Sun Exposed Suprapubic
61.6 TPM
Skin Sun Exposed Lower leg
59.8 TPM
Cólon sigmoide
59.1 TPM
Útero
58.5 TPM
OUTRAS DOENÇAS (2)
epidermolysis bullosa simplex 3, localized or generalized intermediate, with BP230 deficiencyhereditary sensory and autonomic neuropathy type 6
HGNC:1090UniProt:Q03001
RETREG1Reticulophagy regulator 1Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (1)
Dengue virus modulates apoptosis
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory and autonomic, 2B

A form of hereditary sensory and autonomic neuropathy, a genetically and clinically heterogeneous group of disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and by sensory and/or autonomic abnormalities. HSAN2B is an autosomal recessive disorder characterized by impairment of pain, temperature and touch sensation. Onset occurs in the first or second decade, with impaired nociception and progressive mutilating ulceration of the hands and feet with osteomyelitis and acroosteolysis. Amputations of the hands and feet are common. Autonomic dysfunction includes hyperhidrosis, urinary incontinence, and slow pupillary light response.

OUTRAS DOENÇAS (2)
neuropathy, hereditary sensory and autonomic, type 2Bhereditary sensory and autonomic neuropathy type 2
HGNC:25964UniProt:Q9H6L5
ATL3Atlastin-3Disease-causing germline mutation(s) inTolerante
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory, 1F

An autosomal dominant sensory neuropathy affecting the lower limbs. Distal sensory impairment becomes apparent during the second or third decade of life, resulting in painless ulceration of the feet with poor healing, which can progress to osteomyelitis, bone destruction, and amputation. There is no autonomic involvement, spasticity, or cognitive impairment.

OUTRAS DOENÇAS (2)
neuropathy, hereditary sensory, type 1Fhereditary sensory and autonomic neuropathy type 1
HGNC:24526UniProt:Q6DD88
NGFBeta-nerve growth factorDisease-causing germline mutation(s) inRestrito
VIAS BIOLÓGICAS (1)
NGF processing
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory and autonomic, 5

A form of hereditary sensory and autonomic neuropathy, a genetically and clinically heterogeneous group of disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and by sensory and/or autonomic abnormalities. HSAN5 patients manifest loss of pain perception and impaired temperature sensitivity, ulcers, and in some cases self-mutilation. The autonomic involvement is variable.

EXPRESSÃO TECIDUAL(Ubíquo)
Ovário
33.5 TPM
Aorta
30.0 TPM
Fallopian Tube
28.1 TPM
Útero
22.6 TPM
Artéria tibial
21.9 TPM
OUTRAS DOENÇAS (1)
hereditary sensory and autonomic neuropathy type 5
HGNC:7808UniProt:P01138
SPTLC2Serine palmitoyltransferase 2Disease-causing germline mutation(s) inAltamente restrito
VIAS BIOLÓGICAS (1)
Sphingolipid de novo biosynthesis
MECANISMO DE DOENÇA

Neuropathy, hereditary sensory and autonomic, 1C

A form of hereditary sensory and autonomic neuropathy, a genetically and clinically heterogeneous group of disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and by prominent sensory abnormalities with a variable degree of motor and autonomic dysfunction. The neurological phenotype is often complicated by severe infections, osteomyelitis, and amputations. HSAN1C symptoms include loss of touch and vibration in the feet, dysesthesia and severe panmodal sensory loss in the upper and lower limbs, distal lower limb sensory loss with ulceration and osteomyelitis, and distal muscle weakness.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Spinal cord cervical c-1
49.9 TPM
Baço
29.7 TPM
Esôfago - Mucosa
26.9 TPM
Adipose Visceral Omentum
25.0 TPM
Tecido adiposo
24.1 TPM
OUTRAS DOENÇAS (2)
neuropathy, hereditary sensory and autonomic, type 1Chereditary sensory and autonomic neuropathy type 1
HGNC:11278UniProt:O15270
AIFM1Apoptosis-inducing factor 1, mitochondrialDisease-causing germline mutation(s) inAltamente restrito
MECANISMO DE DOENÇA

Combined oxidative phosphorylation deficiency 6

A mitochondrial disease resulting in a neurodegenerative disorder characterized by psychomotor delay, hypotonia, areflexia, muscle weakness and wasting. Some patients manifest prenatal ventriculomegaly and severe postnatal encephalomyopathy.

OUTRAS DOENÇAS (4)
X-linked hereditary sensory and autonomic neuropathy with hearing lossCharcot-Marie-Tooth disease X-linked recessive 4spondyloepimetaphyseal dysplasia, Bieganski typesevere X-linked mitochondrial encephalomyopathy
HGNC:8768UniProt:O95831
KLHL7Kelch-like protein 7Disease-causing germline mutation(s) inTolerante
MECANISMO DE DOENÇA

Perching syndrome

An autosomal recessive multisystem disorder characterized by global developmental delay, dysmorphic facial features, feeding and respiratory difficulties with poor overall growth, axial hypotonia, and joint contractures. The features are variable, even within families, and may also include retinitis pigmentosa, cardiac or genitourinary anomalies, and abnormal sweating.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
28.9 TPM
Cérebro - Hemisfério cerebelar
19.6 TPM
Coração - Ventrículo esquerdo
16.2 TPM
Cerebelo
14.7 TPM
Fibroblastos
14.5 TPM
OUTRAS DOENÇAS (6)
PERCHING syndromeretinitis pigmentosa 42KLHL7-related cold-induced sweating-like syndromeKLHL7-related Bohring-Opitz-like syndrome
HGNC:15646UniProt:Q8IXQ5
KIF1AKinesin-like protein KIF1ADisease-causing germline mutation(s) inAltamente restrito
VIAS BIOLÓGICAS (2)
KinesinsCOPI-dependent Golgi-to-ER retrograde traffic
MECANISMO DE DOENÇA

Spastic paraplegia 30A, autosomal dominant

A form of spastic paraplegia, a neurodegenerative disorder characterized by a slow, gradual, progressive weakness and spasticity of the lower limbs. Rate of progression and the severity of symptoms are quite variable. Initial symptoms may include difficulty with balance, weakness and stiffness in the legs, muscle spasms, and dragging the toes when walking. In some forms of the disorder, bladder symptoms (such as incontinence) may appear, or the weakness and stiffness may spread to other parts of the body. Some SPG30A patients have a pure form of the disorder, limited to spastic paraplegia, whereas others may have a complicated form that includes additional features such as cognitive dysfunction, learning disabilities, peripheral sensorimotor neuropathy, urinary sphincter problems, and/or cerebellar atrophy. SPG30A is characterized by onset in the first or second decades of unsteady spastic gait and hyperreflexia of the lower limbs. Inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Tecido-específico)
Córtex cerebral
331.8 TPM
Brain Frontal Cortex BA9
301.8 TPM
Cerebelo
266.3 TPM
Brain Anterior cingulate cortex BA24
262.4 TPM
Cérebro - Hemisfério cerebelar
235.1 TPM
OUTRAS DOENÇAS (5)
spastic paraplegia 30b, autosomal recessiveneuropathy, hereditary sensory, type 2Cintellectual disability, autosomal dominant 9hereditary sensory and autonomic neuropathy type 2
HGNC:888UniProt:Q12756
NTRK1High affinity nerve growth factor receptorDisease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (1)
TRKA activation by NGF
MECANISMO DE DOENÇA

Congenital insensitivity to pain with anhidrosis

Characterized by a congenital insensitivity to pain, anhidrosis (absence of sweating), absence of reaction to noxious stimuli, self-mutilating behavior, and intellectual disability. This rare autosomal recessive disorder is also known as congenital sensory neuropathy with anhidrosis or hereditary sensory and autonomic neuropathy type IV or familial dysautonomia type II.

EXPRESSÃO TECIDUAL(Tecido-específico)
Próstata
5.7 TPM
Testículo
4.9 TPM
Cervix Endocervix
4.3 TPM
Útero
3.7 TPM
Fallopian Tube
3.5 TPM
OUTRAS DOENÇAS (4)
hereditary sensory and autonomic neuropathy type 4hereditary sensory and autonomic neuropathy type 5familial medullary thyroid carcinomadifferentiated thyroid carcinoma
HGNC:8031UniProt:P04629

Variantes genéticas (ClinVar)

744 variantes patogênicas registradas no ClinVar.

🧬 SCN11A: GRCh37/hg19 3p26.3-14.3(chr3:2263690-55016039)x3 ()
🧬 SCN11A: NM_001349253.2(SCN11A):c.1217A>G (p.Gln406Arg) ()
🧬 SCN11A: NM_001349253.2(SCN11A):c.2102T>C (p.Leu701Pro) ()
🧬 SCN11A: NM_001349253.2(SCN11A):c.1793_1794delinsTA (p.His598Leu) ()
🧬 SCN11A: NM_001349253.2(SCN11A):c.4344del (p.Leu1449fs) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 5.533 variantes classificadas pelo ClinVar.

553
3320
1660
Patogênica (10.0%)
VUS (60.0%)
Benigna (30.0%)
VARIANTES MAIS SIGNIFICATIVAS
DST: DST, HIS269ARG [Pathogenic]
DST: NM_001374736.1:c.905A>G transition in exon 8, resulting in a his302-to-arg (H302... [Pathogenic]
CCT5: NM_012073.5(CCT5):c.521G>A (p.Gly174Asp) [Uncertain significance]
SPTLC1: NM_006415.4(SPTLC1):c.322T>C (p.Phe108Leu) [Uncertain significance]
SPTLC1: NM_006415.4(SPTLC1):c.1324C>G (p.Pro442Ala) [Uncertain significance]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

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Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
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3Fase 35
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·Pré-clínico31
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Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Neuropatia sensitiva e autonômica hereditária

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Selecione um estado ou use sua localização para ver resultados.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Onde estão os ensaios

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27 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

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Publicações mais relevantes

Timeline de publicações
249 papers (10 anos)
#1

Deciphering DST-associated disorders: biallelic variants affecting DST-b cause a congenital myopathy.

Brain : a journal of neurology2026 Feb 07

The dystonin gene (DST) encodes three major isoforms, DST-a, DST-b and DST-e. Biallelic pathogenic variants in DST have previously been associated with two allelic monogenic disorders: hereditary sensory and autonomic neuropathy type VI (caused by a loss of DST-a) and epidermolysis bullosa simplex 3 (caused by a loss of DST-e). We investigated patients diagnosed with congenital myopathy using exome or genome sequencing. In 19 affected individuals from 14 unrelated families, we identified nine different variants in biallelic state located in exons 40-41, specific to DST-b. Affected individuals presented with severe neonatal myopathy characterized by arthrogryposis, hypotonia and dilated cardiomyopathy. Postnatal CPAP ventilation was required in nine patients, and seven died within the first three years of life. Survivors showed an improvement of symptoms, with the oldest three patients, now over 25 years old, exhibiting normal cognition and being ambulatory. RNA analyses demonstrated that transcripts encoding DST-b are predominantly expressed in skeletal muscle, heart tissue and cultured fibroblasts, but not in brain, matching the phenotypic spectrum. Patient-derived fibroblasts exhibited reduced DST mRNA expression. Proteomic analysis confirmed a reduction of DST protein levels due to an absence of the DST-b isoform. Muscle biopsies from four patients aged 1 month to 3 years revealed mild, non-specific myopathic changes. Ultrastructural analysis in three individuals showed mild and focal myofibrillar disruption and non-specific undulating nuclear membranes, with these changes observed in two cases each. Additionally, we identified two homozygous variants affecting both DST-a and DST-b isoforms in four patients from two unrelated families; all presented with severe arthrogryposis and died intrauterine or shortly after birth. Genotype-phenotype correlation in these patients and previously published cases with respective variants resulted in the definition of a DST-associated lethal congenital contracture syndrome. Our findings demonstrate that biallelic variants exclusively affecting DST-b cause an autosomal recessive congenital myopathy. Variants that also impact DST-a besides DST-b result in a more severe, lethal congenital contracture syndrome. The location of the variant within DST allows for phenotype prediction. We propose redefining DST as a disease-associated gene linked to four distinct allelic disease phenotypes.

#2

Long-Term Successful Nonoperative Management of a Displaced Pediatric Odontoid Fracture in Hereditary Sensory and Autonomic Neuropathy Type IV: A Case Report.

JBJS case connector2026 Jan 01

A 6-year-old boy with Hereditary Sensory and Autonomic Neuropathy Type IV (HSAN IV) presented after repetitive self-inflicted head trauma with new-onset headache and neck discomfort. He had a history of self-injurious behavior and was neurologically intact on exam. Imaging revealed a displaced odontoid synchondrosis fracture with C1 to C2 subluxation. Halo immobilization failed due to pin-site complications and behavior. He was successfully managed with staged nonoperative treatment including Minerva casting and cervicothoracic lumbar orthosis bracing. This case highlights the limitations of halo immobilization in patients with pain insensitivity and reports the longest documented follow-up of upper cervical spine injury in HSAN IV.

#3

Exploring the proprioceptive potential of joint receptors using a biomimetic robotic joint.

Scientific reports2026 Feb 03

In neuroscience, joint receptors have traditionally been viewed as limit detectors, providing positional information only at extreme joint angles, while muscle spindles are considered the primary sensors of joint angle position. However, joint receptors are widely distributed throughout the joint capsule, and their full role in proprioception remains unclear. In this study, we specifically focused on mimicking Type I joint receptors, which respond to slow and sustained movements, and quantified their proprioceptive potential using a biomimetic joint developed with robotics technology. Results showed that Type I-like joint receptors alone enabled proprioceptive sensing with an average error of less than 2 degrees in both bending and twisting motions. These findings suggest that joint receptors may play a greater role in proprioception than previously recognized and that the relative contributions of muscle spindles and joint receptors are differentially weighted within neural networks during development and evolution. Furthermore, this work may prompt new discussions on the differential proprioceptive deficits observed between the elbows and knees in patients with hereditary sensory and autonomic neuropathy type III. Together, these findings highlight the potential of biomimetics-based robotic approaches for advancing interdisciplinary research bridging neuroscience, medicine, and robotics.

#4

Clinical and Genetic Characterization of Hereditary Sensory and Autonomic Neuropathy Type IV in a Consanguineous Population: Identification of Novel NTRK1 Variants and Expansion of Phenotypic Spectrum.

American journal of medical genetics. Part A2026 Apr

Hereditary sensory and autonomic neuropathy type IV (HSAN4) is a rare neurological disorder characterized by anhidrosis and congenital insensitivity to pain caused by biallelic pathogenic variants in NTRK1. The condition develops because of dorsal root and autonomic ganglion neurodegeneration, which ultimately results in reduced sensation and autonomic neurological dysfunction. We ascertained several neuropathic patients and performed genetic testing using gene panels and exome sequencing (ES). Genetic variants were confirmed by Sanger sequencing. Thirteen families, each with a single affected individual, participated in this study. Genetic testing revealed that all patients carried disease-causing variants in NTRK1. We identified seven different variants within our cohort, including two novel variants (c.1922T>C:p.Leu641Pro and c.1071_1072insTGCC:p.Asn358Cysfs*45). While some variants suggest a possible founder effect, the identification of new variants reflects the genetic diversity within the Saudi population. In addition to the cardinal clinical feature of HSAN4, patients exhibited various other symptoms like motor difficulties, microcephaly, recurrent hip dislocation, dystrophic nails, hypotrichosis, and various dysmorphic features. This study provides clinical information on a large number of patients, updates the prevalence and epidemiologic data in our population, and further expands the understanding of the disease's genetic and clinical spectrum within a highly consanguineous population.

#5

Type IV Hereditary Sensory and Autonomic Neuropathy: Challenges and Functional Outcomes.

Journal of pediatric orthopedics2026 Mar 01

Type IV Hereditary Sensory and Autonomic Neuropathy (HSAN IV) is an exceedingly rare autosomal recessive disease classically characterized by generalized loss of temperature/pain sensation, intellectual disability, and anhidrosis. HSAN IV patients are subject to repeated orthopaedic injuries and complications. There is some data on the clinical presentation, but limited data on the diagnostic and treatment challenges, and functional outcomes with HSAN IV. This is a retrospective case series of 4 siblings with HSAN IV. Medical records were reviewed for data regarding presentation, diagnosis, and management. Functional outcomes were assessed using the Pediatric Outcomes Data Collection Instrument, with historical normative data used as a control. The 2 populations were evaluated with a Welch-modified 2-sample t test to explore the alternative hypothesis that HSAN IV patients are significantly functionally impaired. Our study included 4 siblings, 2 males and 2 females, aged 3, 8, 12, and 21 years, respectively. The average time to diagnosis was 53 months, and each diagnosis was confirmed through whole-exome sequencing. The challenges faced included delayed diagnosis, diverse clinical presentations, unestablished treatment guidelines, and unnecessary healthcare utilization before definitive diagnosis. The comparison of means between the normative population and the HSAN IV patients revealed a significant difference in sports/play ( T -score =-4.14, P =0.025) and global function ( T -score =-3.19, P =0.049) but no significant difference in upper extremity function ( P =0.216), transfer mobility ( P =0.164), pain/comfort ( P =0.955), and happiness ( P =0.995). HSAN IV is a rare genetic condition with protean clinical manifestations that can result in significant deficits in play and global function. The clinical manifestations are varied, including multiple fractures, delayed/abnormal fracture healing, bone deformity, osteomyelitis, and various other non-orthopaedic manifestations. Whole-exome sequencing is a useful tool that, when combined with a high index of clinical suspicion, can expedite the diagnostic process for HSAN IV. Patients can benefit from earlier diagnosis and earlier access to education to prevent functional deterioration and repeat medical interventions. IV-case series.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC157 artigos no totalmostrando 199

2026

Long-Term Successful Nonoperative Management of a Displaced Pediatric Odontoid Fracture in Hereditary Sensory and Autonomic Neuropathy Type IV: A Case Report.

JBJS case connector
2026

Exploring the proprioceptive potential of joint receptors using a biomimetic robotic joint.

Scientific reports
2026

Clinical and Genetic Characterization of Hereditary Sensory and Autonomic Neuropathy Type IV in a Consanguineous Population: Identification of Novel NTRK1 Variants and Expansion of Phenotypic Spectrum.

American journal of medical genetics. Part A
2025

Hereditary Sensory and Autonomic Neuropathy Type 2: A Case Report and a Review of the Literature.

Brain sciences
2025

The neuropathy-linked protein TECPR2 is a Rab5 effector that regulates cargo recycling from early endosomes.

Nature communications
2025

A novel homozygous DST variant causes hereditary sensory and autonomic neuropathy in a Pakistani family.

Human genome variation
2025

Oral Rehabilitation in Patient With Hereditary Sensory and Autonomic Neuropathy (HSAN) Type V: Clinical Report.

Case reports in dentistry
2025

Neuropathy-associated Tecpr2 mutation knock-in mice reveal endolysosomal loss of function phenotypes in neurons and microglia.

Cell death & disease
2025

Congenital Insensitivity to Pain Syndrome With Hidrosis in a 17-Year-Old Female.

Cureus
2025

FAM134B in Cellular Homeostasis: Bridging Endoplasmic Reticulum-Phagy to Human Diseases.

International journal of biological sciences
2025

Rare autosomal recessive hereditary sensory and autonomic neuropathy type VI in a Pakistani family caused by a novel DST variant.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2025

Hummingbird sign in a patient with DNMT1-related disorder.

Neurocase
2025

Lenticulostriate vasculopathy in newborns: whole genome sequencing data analysis.

Frontiers in pediatrics
2025

Characterization of novel and recurrent SPTLC2 variants in childhood-onset amyotrophic lateral sclerosis: Insights into sphingolipid dysregulation.

Journal of neuromuscular diseases
2026

Type IV Hereditary Sensory and Autonomic Neuropathy: Challenges and Functional Outcomes.

Journal of pediatric orthopedics
2025

Congenital Insensitivity to Pain With Anhidrosis: First Reported Case in Nepal.

Clinical case reports
2025

Advances in the treatment of familial dysautonomia: what does the future hold?

Expert review of neurotherapeutics
2026

Deciphering DST-associated disorders: biallelic variants affecting DST-b cause a congenital myopathy.

Brain : a journal of neurology
2025

SCN9A should not be considered an epilepsy gene; Refuting a gene-disease association.

Epilepsia
2025

[Peripheral Nerve: Cause and Pathology of Inherited Peripheral Neuropathy].

Brain and nerve = Shinkei kenkyu no shinpo
2025

Recurrent Osteomyelitis in a Paediatric Patient with a Novel NTRK1 Mutation: A Case Report on Congenital Insensitivity to Pain with Anhidrosis.

Children (Basel, Switzerland)
2025

Effect of SPTLC1 on type 2 diabetes mellitus.

World journal of diabetes
2025

First instance of pain in congenital pain insensitivity with anhidrosis.

Clinical neurology and neurosurgery
2025

Blended phenotype of TECPR2-associated hereditary sensory-autonomic neuropathy and Temple syndrome.

Annals of clinical and translational neurology
2025

A novel treatment strategy with hyperbaric oxygen of chronic osteomyelitis and pseudoarthrosis in a child with congenital hereditary sensory and autonomic neuropathy type 4 congenital insensitivity to pain with anhidrosis syndrome: a case report.

Journal of medical case reports
2025

A novel mutation in the WNK1/HSN2 gene causing hereditary sensory and autonomic neuropathy type 2 in Chinese patient.

Journal of human genetics
2024

Genetic and functional analyses of SPTLC1 in juvenile amyotrophic lateral sclerosis.

Journal of neurology
2024

Novel TECPR2 variant in two cases of hereditary sensory and autonomic neuropathy type 9: insights from genetic characterization and comprehensive literature review.

BMC neurology
2024

Atypical Presentation of Congenital Insensitivity to Pain With Anhidrosis Leading to Diagnostic Odyssey.

Molecular genetics & genomic medicine
2024

A Novel Pathogenic Mutation in WNK1 Gene Causing HSAN Type II in Three Siblings.

Journal of molecular neuroscience : MN
2025

Congenital Insensitivity to Pain With Anhidrosis Is Associated With Harlequin Color Change: A Survey Study.

Pediatric dermatology
2024

A RETREG1 variant is associated with hereditary sensory and autonomic neuropathy with acral self-mutilation in purebred German Spitz.

Animal genetics
2024

Hereditary Sensory and Autonomic Neuropathy-Report of Two Cases in Siblings and Review of Literature.

Indian journal of dermatology
2024

Management Targeted Genetic Evaluation of an Idiopathic Neuropathy Cohort Through ATTRv Amyloidosis Screening.

HCA healthcare journal of medicine
2024

Development of a Multiplexed Sphingolipids Method for Diagnosis of Inborn Errors of Ceramide Metabolism.

Clinical chemistry
2024

Sensory-motor circuit is a therapeutic target for dystonia musculorum mice, a model of hereditary sensory and autonomic neuropathy 6.

Science advances
2024

Fam134c and Fam134b shape axonal endoplasmic reticulum architecture in vivo.

EMBO reports
2024

Functional and Molecular Characterization of New SPTLC1 Missense Variants in Patients with Hereditary Sensory and Autonomic Neuropathy Type 1 (HSAN1).

Genes
2024

A novel NTRK1 splice site variant causing congenital insensitivity to pain with anhidrosis in a Chinese family.

Frontiers in genetics
2024

Serine Palmitoyltransferase (SPT)-related Neurodegenerative and Neurodevelopmental Disorders.

Journal of neuromuscular diseases
2024

Identification of a Novel Homozygous Mutation in PRDM12 Gene in a Patient with Hereditary Sensory and Autonomic Neuropathy Type VIII.

Archives of Iranian medicine
2024

A rare case of congenital insensitivity to pain with anhidrosis.

Paediatrics and international child health
2024

Clinical and genetic characteristics of three patients with congenital insensitivity to pain with anhidrosis: Case reports and a review of the literature.

Molecular genetics & genomic medicine
2024

[Hereditary sensory and autonomic neuropathy 1E showing hyperreflexia: a case report].

Rinsho shinkeigaku = Clinical neurology
2024

TECPR2-related hereditary sensory and autonomic neuropathy in two siblings from Palestine.

American journal of medical genetics. Part A
2024

DNMT1 Y495C mutation interferes with maintenance methylation of imprinting control regions.

The international journal of biochemistry & cell biology
2024

Phenotypes of a toddler with hereditary sensory and autonomic neuropathy type IV: comparing with normal: A case report.

Medicine
2023

Exploring CNS Involvement in Pain Insensitivity in Hereditary Sensory and Autonomic Neuropathy Type 4: Insights from Tc-99m ECD SPECT Imaging.

Tomography (Ann Arbor, Mich.)
2023

Congenital Insensitivity to Pain with Anhidrosis: A Case Report.

Cureus
2023

Mfsd7b facilitates choline transport and missense mutations affect choline transport function.

Cellular and molecular life sciences : CMLS
2024

Recurrent de novo SPTLC2 variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesis.

Journal of neurology, neurosurgery, and psychiatry
2023

Expression pattern analysis and characterization of the hereditary sensory and autonomic neuropathy 2 A (HSAN2A) gene with no lysine kinase (WNK1) in human dorsal root ganglion.

Experimental neurology
2023

Multisystemic RFC1-Related Disorder: Expanding the Phenotype Beyond Cerebellar Ataxia, Neuropathy, and Vestibular Areflexia Syndrome.

Neurology. Clinical practice
2023

A novel HSPB1S139F mouse model of Charcot-Marie-Tooth Disease.

Prostaglandins & other lipid mediators
2024

Roles of dystonin isoforms in the maintenance of neural, muscle, and cutaneous tissues.

Anatomical science international
2023

Autonomic failure: Clinicopathologic, physiologic, and genetic aspects.

Handbook of clinical neurology
2023

DST variants are responsible for neurogenic arthrogryposis multiplex congenita enlarging the spectrum of type VI hereditary sensory autonomic neuropathy.

Clinical genetics
2023

Orthopedic Manifestations of Hereditary Sensory and Autonomic Neuropathy IV in a 10-Year-Old Patient.

The Iowa orthopaedic journal
2023

SPTLC1 p.Leu38Arg, a novel mutation associated with childhood ALS.

Biochimica et biophysica acta. Molecular and cell biology of lipids
2023

Molecular characterization of wild-type and HSAN2B-linked FAM134B.

Molecular biology reports
2023

Expanding the Knowledge of KIF1A-Dependent Disorders to a Group of Polish Patients.

Genes
2023

Genetic predisposition to ocular surface disorders and opportunities for gene-based therapies.

The ocular surface
2023

Specific Deoxyceramide Species Correlate with Expression of Macular Telangiectasia Type 2 (MacTel2) in a SPTLC2 Carrier HSAN1 Family.

Genes
2024

Sensorimotor control in the congenital absence of functional muscle spindles.

Experimental physiology
2023

The NGF R100W Mutation, Associated with Hereditary Sensory Autonomic Neuropathy Type V, Specifically Affects the Binding Energetic Landscapes of NGF and of Its Precursor proNGF and p75NTR.

Biology
2023

Spectrum of SPTLC1-related disorders: a novel case of 'Ser331 syndrome' that expand the phenotype of hereditary sensory and autonomic neuropathy type 1A and motor neuron diseases.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2023

A rare case of hereditary sensory and autonomic neuropathy type II.

Clinical case reports
2023

Spatial proteomics reveals secretory pathway disturbances caused by neuropathy-associated TECPR2.

Nature communications
2023

Novel de novo DNMT1 gene mutation associated with hereditary sensory and autonomic neuropathy 1E (HSAN1E).

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2022

Isolation and transfection of myenteric neurons from mice for patch-clamp applications.

Frontiers in molecular neuroscience
2023

NGF and BDNF in pediatrics syndromes.

Neuroscience and biobehavioral reviews
2023

Human TrkAR649W mutation impairs nociception, sweating and cognitive abilities: a mouse model of HSAN IV.

Human molecular genetics
2022

Congenital Insensitivity to Pain With Anhidrosis: A Case Report and Review of the Pertinent Literature.

Cureus
2023

Impact of the coronavirus pandemic on families of patients with congenital insensitivity to pain with anhidrosis.

Pediatrics international : official journal of the Japan Pediatric Society
2022

Cough as a neurological sign: What a clinician should know.

World journal of critical care medicine
2023

Identification of sensory dysfunction and nervous structure changes in Fam134b knockout mice.

Neurological research
2022

Association of variants in the KIF1A gene with amyotrophic lateral sclerosis.

Translational neurodegeneration
2024

KIF1A novel frameshift variant p.(Ser887Profs*64) exhibits clinical heterogeneity in a Pakistani family with hereditary sensory and autonomic neuropathy type IIC.

The International journal of neuroscience
2022

A de novo c.113 T > C: p.L38R mutation of SPTLC1: case report of a girl with sporadic juvenile amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis & frontotemporal degeneration
2022

Anesthetic management of a child with congenital insensitivity to pain with anhidrosis: A case report.

Frontiers in surgery
2022

WNK1/HSN2 mediates neurite outgrowth and differentiation via a OSR1/GSK3β-LHX8 pathway.

Scientific reports
2022

Multi-type RFC1 repeat expansions as the most common cause of hereditary sensory and autonomic neuropathy.

Frontiers in neurology
2022

Isoform-specific mutation in Dystonin-b gene causes late-onset protein aggregate myopathy and cardiomyopathy.

eLife
2022

Hereditary Sensory and Autonomic Neuropathy Type II in a Female Child with Multiple Orthopaedic Ailments: Diagnosis and Operative Management.

Indian journal of orthopaedics
2022

SPTLC1 variants associated with ALS produce distinct sphingolipid signatures through impaired interaction with ORMDL proteins.

The Journal of clinical investigation
2022

Keratoconus associated with hereditary sensory and autonomic neuropathy II.

Indian journal of ophthalmology
2022

A case report: Anesthetic management for open-heart surgery in a child with congenital insensitivity to pain with anhidrosis.

Paediatric anaesthesia
2022

NTRK1-related Hereditary Sensory and Autonomic Neuropathy Type 4: The Role of the Histamine Challenge Test.

Child neurology open
2022

Genetic pain loss disorders.

Nature reviews. Disease primers
2022

New Insights into the Neuromyogenic Spectrum of a Gain of Function Mutation in SPTLC1.

Genes
2022

Novel RETREG1 (FAM134B) founder allele is linked to HSAN2B and renal disease in a Turkish family.

American journal of medical genetics. Part A
2022

Hereditary Sensory and Autonomic Neuropathy Type VIII: Congenital Insensitivity to Pain with Anhidrosis.

Indian dermatology online journal
2022

Pathogenic DST sequence variants result in either epidermolysis bullosa simplex (EBS) or hereditary sensory and autonomic neuropathy type 6 (HSAN-VI).

Experimental dermatology
2022

Hereditary Sensory and Autonomic Neuropathy: A Case Series of Six Children.

Neurology India
2022

Hereditary Autonomic Neuropathy of the Oral Cavity and its Management.

Iranian journal of child neurology
2022

Pregnancy in hereditary sensory and autonomic neuropathy type V: A case report and literature review.

Taiwanese journal of obstetrics & gynecology
2022

A neuropathy-associated kinesin KIF1A mutation hyper-stabilizes the motor-neck interaction during the ATPase cycle.

The EMBO journal
2022

A novel variant in the dystonin gene causing hereditary sensory autonomic neuropathy type VI in a male infant: Case report and literature review.

American journal of medical genetics. Part A
2022

Developmental regulation of neuronal gene expression by Elongator complex protein 1 dosage.

Journal of genetics and genomics = Yi chuan xue bao
2022

Precision mouse models of Yars/dominant intermediate Charcot-Marie-Tooth disease type C and Sptlc1/hereditary sensory and autonomic neuropathy type 1.

Journal of anatomy
2022

1-Deoxysphinganine initiates adaptive responses to serine and glycine starvation in cancer cells via proteolysis of sphingosine kinase.

Journal of lipid research
2021

A 5-Year-Old Palestinian Bedouin Girl with Repeated Self-Induced Injuries to the Digits, a Diagnosis of Congenital Insensitivity to Pain, and Anhidrosis.

The American journal of case reports
2021

Generation of a transgenic mouse embryonic stem cell line expressing Dnmt1Y495C mutation associated with HSAN1E disorder.

Stem cell research
2021

Not Another Case Of Juvenile Idiopathic Arthritis: Congenital Insensitivity To Pain Presenting With Joint Problems.

Journal of Ayub Medical College, Abbottabad : JAMC
2021

Hereditary sensory and autonomic neuropathy in a family of mixed breed dogs associated with a novel RETREG1 variant.

Journal of veterinary internal medicine
2021

Towards personalized medicine for amyotrophic lateral sclerosis.

Trends in endocrinology and metabolism: TEM
2021

Genotype and phenotype distribution of 435 patients with Charcot-Marie-Tooth disease from central south China.

European journal of neurology
2021

Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis.

Nature medicine
2021

Understanding pain perception through genetic painlessness diseases: The role of NGF and proNGF.

Pharmacological research
2021

Late-onset hereditary sensory and autonomic neuropathy type 2B caused by novel compound heterozygous mutations in FAM134B presenting as chronic recurrent ulcers on the soles.

Indian journal of dermatology, venereology and leprology
2021

The long chain base unsaturation has a stronger impact on 1-deoxy(methyl)-sphingolipids biophysical properties than the structure of its C1 functional group.

Biochimica et biophysica acta. Biomembranes
2021

Expanding the Genotypic Spectrum of Congenital Sensory and Autonomic Neuropathies Using Whole-Exome Sequencing.

Neurology. Genetics
2021

Clinical, neuroimaging, and molecular spectrum of TECPR2-associated hereditary sensory and autonomic neuropathy with intellectual disability.

Human mutation
2021

AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.

Trends in molecular medicine
2021

Cerebellar Ataxia as a Common Clinical Presentation Associated with DNMT1 p.Y511H and a Review of the Literature.

Journal of molecular neuroscience : MN
2021

[A case report of hereditary sensory and autonomic neuropathy type Ⅶ].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2021

Reduced Myocardial Uptake of 123I-MIBG in Congenital Insensitivity to Pain With Anhidrosis.

Clinical nuclear medicine
2021

Frequency and burden of gastrointestinal symptoms in familial dysautonomia.

Clinical autonomic research : official journal of the Clinical Autonomic Research Society
2021

Bilateral harlequin syndrome, unilateral Horner syndrome, and Riga-Fede disease as presenting features of hereditary sensory and autonomic neuropathy type IV.

Pediatric dermatology
2020

High prevalence of carpal tunnel syndrome in individuals with rare nerve growth factor-beta mutation.

Brain communications
2021

1-Deoxysphingolipids cause autophagosome and lysosome accumulation and trigger NLRP3 inflammasome activation.

Autophagy
2020

Sisters with No Pain, No Tears: A Report of a New Variant of Hereditary Sensory and Autonomic Neuropathy (Type IX) Caused by a Novel SCN11A Mutation.

Indian journal of dermatology
2021

Heterozygous KIF1A variants underlie a wide spectrum of neurodevelopmental and neurodegenerative disorders.

Journal of medical genetics
2020

Diverse dystonin gene mutations cause distinct patterns of Dst isoform deficiency and phenotypic heterogeneity in Dystonia musculorum mice.

Disease models & mechanisms
2020

Elbow proprioception is normal in patients with a congenital absence of functional muscle spindles.

The Journal of physiology
2020

Disruption of dystonin in Schwann cells results in late-onset neuropathy and sensory ataxia.

Glia
2020

Canonical and 1-Deoxy(methyl) Sphingoid Bases: Tackling the Effect of the Lipid Structure on Membrane Biophysical Properties.

Langmuir : the ACS journal of surfaces and colloids
2020

Retrograde nerve growth factor signaling abnormalities in familial dysautonomia.

The Journal of clinical investigation
2020

A Rare KIF1A Missense Mutation Enhances Synaptic Function and Increases Seizure Activity.

Frontiers in genetics
2022

Population Study of Hand and Wrist Manifestations of Congenital Insensitivity to Pain.

Hand (New York, N.Y.)
2020

A novel biallelic single base insertion in WNK1 in a Pakistani family with congenital insensitivity to pain.

Journal of human genetics
2020

HSAN-VI: A spectrum disorder based on dystonin isoform expression.

Neurology. Genetics
2020

Cataplexy and ataxia: red flags for the diagnosis of DNA methyltransferase 1 mutation.

Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine
2020

FAM134B oligomerization drives endoplasmic reticulum membrane scission for ER-phagy.

The EMBO journal
2020

Characterization of gastrointestinal pathologies in the dystonia musculorum mouse model for hereditary sensory and autonomic neuropathy type VI.

Neurogastroenterology and motility
2020

First Portuguese patient presenting with hereditary sensory and autonomic neuropathy type 1E associated with a novel mutation in DNMT1 gene.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2019

Hereditary Sensory and Autonomic Neuropathy 2B Caused by a Novel RETREG1 Mutation (c.765dupT) and Paternal Uniparental Isodisomy of Chromosome 5.

Frontiers in genetics
2019

The NGFR100W Mutation Specifically Impairs Nociception without Affecting Cognitive Performance in a Mouse Model of Hereditary Sensory and Autonomic Neuropathy Type V.

The Journal of neuroscience : the official journal of the Society for Neuroscience
2019

Focal Epithelial Hyperplasia in a Child with Hereditary Sensory and Autonomic Neuropathy type IV: A Rare Co-Occurrence.

Journal of dentistry for children (Chicago, Ill.)
2019

Serine and Lipid Metabolism in Macular Disease and Peripheral Neuropathy.

The New England journal of medicine
2019

Cytotoxicity of 1-deoxysphingolipid unraveled by genome-wide genetic screens and lipidomics in Saccharomyces cerevisiae.

Molecular biology of the cell
2019

De novo pathogenic DNM1L variant in a patient diagnosed with atypical hereditary sensory and autonomic neuropathy.

Molecular genetics & genomic medicine
2019

Congenital Insensitivity to Pain with Anhidrosis: A Case with Self-Inflicted Oral Ulcerations.

Journal of dentistry for children (Chicago, Ill.)
2019

A Model of Hereditary Sensory and Autonomic Neuropathy Type 1 Reveals a Role of Glycosphingolipids in Neuronal Polarity.

The Journal of neuroscience : the official journal of the Society for Neuroscience
2019

The coexistence of a novel WNK1 variant and a copy number variation causes hereditary sensory and autonomic neuropathy type IIA.

BMC medical genetics
2019

A Novel Variant (Asn177Asp) in SPTLC2 Causing Hereditary Sensory Autonomic Neuropathy Type 1C.

Neuromolecular medicine
2019

DNMT1-complex disorder caused by a novel mutation associated with an overlapping phenotype of autosomal-dominant cerebellar ataxia, deafness, and narcolepsy (ADCA-DN) and hereditary sensory neuropathy with dementia and hearing loss (HSN1E).

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2019

Chemoreflex failure and sleep-disordered breathing in familial dysautonomia: Implications for sudden death during sleep.

Autonomic neuroscience : basic & clinical
2019

Hereditary sensory and autonomic neuropathy type IC accompanied by upper motor neuron abnormalities and type II juxtafoveal retinal telangiectasias.

Journal of the peripheral nervous system : JPNS
2018

Extreme Ends of Pain Sensitivity in SCN9A Mutation Variants: Case Report and Literature Review.

Innovations in clinical neuroscience
2019

ATL3 Is a Tubular ER-Phagy Receptor for GABARAP-Mediated Selective Autophagy.

Current biology : CB
2020

Heterotopic ossifications and Charcot joints: Congenital insensitivity to pain with anhidrosis (CIPA) and a novel NTRK1 gene mutation.

European journal of medical genetics
2019

Uncoupling neurotrophic function from nociception of nerve growth factor: what can be learned from a rare human disease?

Neural regeneration research
2019

Randomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1.

Neurology
2019

Plantar Ulcers and Neuropathic Arthropathies: Associated Diseases, Polyneuropathy Correlates, and Risk Covariates.

Advances in skin & wound care
2019

Cholinergic striatal neurons are increased in HSAN V homozygous mice despite reduced NGF bioavailability.

Biochemical and biophysical research communications
2019

Comprehensive Computational Analysis of Protein Phenotype Changes Due to Plausible Deleterious Variants of Human SPTLC1 Gene.

International journal of molecular and cellular medicine
2019

The novel de novo mutation of KIF1A gene as the cause for Spastic paraplegia 30 in a Japanese case.

eNeurologicalSci
2018

A novel nonsense mutation in WNK1/HSN2 associated with sensory neuropathy and limb destruction in four siblings of a large Iranian pedigree.

BMC neurology
2018

V144D Mutation of SPTLC1 Can Present with Both Painful and Painless Phenotypes in Hereditary Sensory and Autonomic Neuropathies Type I.

Case reports in genetics
2019

Painless Nerve Growth Factor: A TrkA biased agonist mediating a broad neuroprotection via its actions on microglia cells.

Pharmacological research
2018

Generation of the human induced pluripotent stem cell line UKWNLi002-A from dermal fibroblasts of a woman with a heterozygous c.608 C>T (p.Thr203Met) mutation in exon 3 of the nerve growth factor gene potentially associated with hereditary sensory and autonomic neuropathy type 5.

Stem cell research
2018

Molecular diagnosis of inherited peripheral neuropathies by targeted next-generation sequencing: molecular spectrum delineation.

BMJ open
2018

Identification of a novel DNMT1 mutation in a Chinese patient with hereditary sensory and autonomic neuropathy type IE.

BMC neurology
2019

Sensory neuropathy-causing mutations in ATL3 affect ER-mitochondria contact sites and impair axonal mitochondrial distribution.

Human molecular genetics
2019

Resting Energy Expenditure in Patients With Familial Dysautonomia: A Preliminary Study.

Journal of pediatric gastroenterology and nutrition
2018

A third HSAN5 mutation disrupts the nerve growth factor furin cleavage site.

Molecular pain
2018

Office-Based Anesthetic and Oral Surgical Management of a Child With Hereditary Sensory Autonomic Neuropathy Type IV: A Case Report.

Anesthesia progress
2018

Impaired sensorimotor control of the hand in congenital absence of functional muscle spindles.

Journal of neurophysiology
2018

Identification of a novel mutation of the NTRK1 gene in patients with congenital insensitivity to pain with anhidrosis (CIPA).

Gene
2018

Hereditary sensory and autonomic neuropathy in a male child: 'The other side of not feeling pain'.

BMJ case reports
2018

Tsc3 regulates SPT amino acid choice in Saccharomyces cerevisiae by promoting alanine in the sphingolipid pathway.

Journal of lipid research
2019

A Child Presenting with Recurrent Corneal Ulcers: Hereditary Sensory and Autonomic Neuropathy IV (HSAN IV).

Neuro-ophthalmology (Aeolus Press)
2018

Congenital Loss of Permanent Teeth in a Patient With Congenital Insensitivity to Pain With Anhidrosis due to 2 Novel Mutations in the NTRK1 Gene.

Journal of oral and maxillofacial surgery : official journal of the American Association of Oral and Maxillofacial Surgeons
2018

Late-onset hereditary sensory and autonomic neuropathy expands the phenotypic spectrum of MFN2-related diseases.

Neuropathology : official journal of the Japanese Society of Neuropathology
2018

Dystonin-A3 upregulation is responsible for maintenance of tubulin acetylation in a less severe dystonia musculorum mouse model for hereditary sensory and autonomic neuropathy type VI.

Human molecular genetics
2018

Midface toddler excoriation syndrome (MiTES) can be caused by autosomal recessive biallelic mutations in a gene for congenital insensitivity to pain, PRDM12.

The British journal of dermatology
2018

Sensory-Neuropathy-Causing Mutations in ATL3 Cause Aberrant ER Membrane Tethering.

Cell reports
2018

Repeated hyperhidrosis and chilblain-like swelling with ulceration of the fingers and toes in hereditary sensory and autonomic neuropathy type II.

The Journal of dermatology
2018

Cerebellar ataxia, neuropathy, vestibular areflexia syndrome (CANVAS) with chronic cough and preserved muscle stretch reflexes: evidence for selective sparing of afferent Ia fibres.

Journal of neurology
2018

Hereditary Sensory and Autonomic Neuropathy Presenting With Mutilating Trophic Ulcers.

Wounds : a compendium of clinical research and practice
2018

Glycogen synthase kinase 3ß functions as a positive effector in the WNK signaling pathway.

PloS one
2018

Swedish Nerve Growth Factor Mutation (NGFR100W) Defines a Role for TrkA and p75NTR in Nociception.

The Journal of neuroscience : the official journal of the Society for Neuroscience
2018

Founder mutation in IKBKAP gene causes vestibular impairment in familial dysautonomia.

Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology
2018

RETREG1 (FAM134B): A new player in human diseases: 15 years after the discovery in cancer.

Journal of cellular physiology
2017

[A case of hereditary sensory and autonomic neuropathy type 1E with frontal lobe dysfunction as an initial symptom].

Rinsho shinkeigaku = Clinical neurology
2018

Anesthetic Management of a Patient With De Novo Hereditary Sensory and Autonomic Neuropathy, Type VII: A Case Report.

A&A practice
2018

Motoneuron degeneration in the trigeminal motor nucleus innervating the masseter muscle in Dystonia musculorum mice.

Neurochemistry international
2017

Clinical and metabolic consequences of L-serine supplementation in hereditary sensory and autonomic neuropathy type 1C.

Cold Spring Harbor molecular case studies
2017

Riley-Day Syndrome in a Hispanic Infant of Non-Jewish Ashkenazi Descent.

Journal of clinical and diagnostic research : JCDR
2017

Oral manifestations, dental management, and a rare homozygous mutation of the PRDM12 gene in a boy with hereditary sensory and autonomic neuropathy type VIII: a case report and review of the literature.

Journal of medical case reports
2017

BPAG1 in muscles: Structure and function in skeletal, cardiac and smooth muscle.

Seminars in cell & developmental biology
2017

Animal and cellular models of familial dysautonomia.

Clinical autonomic research : official journal of the Clinical Autonomic Research Society
2016

[ORO-DENTO-FACIAL MANIFESTATIONS IN PATIENTS WITH FAMILIAL DYSAUTONOMIA].

Harefuah
2017

Sudden Unexpected Death During Sleep in Familial Dysautonomia: A Case-Control Study.

Sleep
2017

BGP-15 prevents the death of neurons in a mouse model of familial dysautonomia.

Proceedings of the National Academy of Sciences of the United States of America
2017

WNK1/HSN2 founder mutation in patients with hereditary sensory and autonomic neuropathy: A Japanese cohort study.

Clinical genetics
2017

Identification of a novel nonsense mutation of the neurotrophic tyrosine kinase receptor type 1 gene in two siblings with congenital insensitivity to pain with anhidrosis.

The Journal of international medical research
2017

Exome sequencing identifies novel NTRK1 mutations in patients with HSAN-IV phenotype.

American journal of medical genetics. Part A

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Doença com base genética

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Conselhos e sociedades

Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

Perguntas frequentes

O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu

Respostas geradas por IA a partir das fontes citadas

A condição é causada por modificações genéticas hereditárias que afetam o funcionamento dos neurônios sensoriais periféricos. Mutações em genes como SCN9A, SCN11A, ATL1 e SPTLC1 estão entre os fatores identificados.

Referências

Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete

7 publicações do grafo RarasNet (PubMed) · 6 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.

  1. ORPHA:140471
    Orphanet
  2. GARD:12688
    GARD (NIH)
  3. Q3702898
    Wikidata

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Citar este verbete

Raras. (s.d.). Neuropatia sensitiva e autonômica hereditária. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/neuropatia-sensitiva-e-autonomica-hereditaria

Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Compêndio · Raras BR

Neuropatia sensitiva e autonômica hereditária

ORPHA:140471 · MONDO:0015364
CID-11
Ensaios
42 ativos
MedGen
UMLS
C0027889
EuropePMC
Wikidata
Papers 10a

📋 Origem dos dados

Esta página agrega dados de fontes públicas e oficiais. Dados sobre cobertura no SUS (PCDT, CEAF) são verificados ativamente por agente proativo (ver badge no infobox). Demais dados têm atribuição de fonte + data da última sincronização — clique para abrir o original.

Doença rara (ontologia)
fonte: Orphanet
Identificador unificado
fonte: MONDO
Indexação biomédica
fonte: MeSH (NLM)
Dado público estruturado
fonte: Wikidata
Rara