Um caso de neuropatia periférica sensorial causada por uma modificação hereditária do genoma do indivíduo.
Introdução
O que você precisa saber de cara
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), traduzidoUm caso de neuropatia periférica sensorial causada por uma modificação hereditária do genoma do indivíduo.
Encontrou um erro ou informação desatualizada? Sugira uma correção →
Entender a doença
Do básico ao detalhe, leia no seu ritmo
Preparando trilha educativa...
Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 118 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 279 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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 oficiaisNeuropathy, 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.
Primary erythermalgia
Autosomal dominant disease characterized by recurrent episodes of severe pain associated with redness and warmth in the feet or hands.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Variantes genéticas (ClinVar)
744 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 5.533 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
44 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Neuropatia sensitiva e autonômica hereditária
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
Ensaios em destaque
Com ensaio aberto em 20 países. O ponto verde marca onde há vaga agora.
🟢 Recrutando agora
27 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.
Outros ensaios clínicos
170 ensaios clínicos encontrados, 42 ativos.
Publicações mais relevantes
Deciphering DST-associated disorders: biallelic variants affecting DST-b cause a congenital myopathy.
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.
Long-Term Successful Nonoperative Management of a Displaced Pediatric Odontoid Fracture in Hereditary Sensory and Autonomic Neuropathy Type IV: A Case Report.
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.
Exploring the proprioceptive potential of joint receptors using a biomimetic robotic joint.
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.
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.
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.
Type IV Hereditary Sensory and Autonomic Neuropathy: Challenges and Functional Outcomes.
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
Long-Term Successful Nonoperative Management of a Displaced Pediatric Odontoid Fracture in Hereditary Sensory and Autonomic Neuropathy Type IV: A Case Report.
Exploring the proprioceptive potential of joint receptors using a biomimetic robotic joint.
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.
Hereditary Sensory and Autonomic Neuropathy Type 2: A Case Report and a Review of the Literature.
The neuropathy-linked protein TECPR2 is a Rab5 effector that regulates cargo recycling from early endosomes.
📚 EuropePMC157 artigos no totalmostrando 199
Long-Term Successful Nonoperative Management of a Displaced Pediatric Odontoid Fracture in Hereditary Sensory and Autonomic Neuropathy Type IV: A Case Report.
JBJS case connectorExploring the proprioceptive potential of joint receptors using a biomimetic robotic joint.
Scientific reportsClinical 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 AHereditary Sensory and Autonomic Neuropathy Type 2: A Case Report and a Review of the Literature.
Brain sciencesThe neuropathy-linked protein TECPR2 is a Rab5 effector that regulates cargo recycling from early endosomes.
Nature communicationsA novel homozygous DST variant causes hereditary sensory and autonomic neuropathy in a Pakistani family.
Human genome variationOral Rehabilitation in Patient With Hereditary Sensory and Autonomic Neuropathy (HSAN) Type V: Clinical Report.
Case reports in dentistryNeuropathy-associated Tecpr2 mutation knock-in mice reveal endolysosomal loss of function phenotypes in neurons and microglia.
Cell death & diseaseCongenital Insensitivity to Pain Syndrome With Hidrosis in a 17-Year-Old Female.
CureusFAM134B in Cellular Homeostasis: Bridging Endoplasmic Reticulum-Phagy to Human Diseases.
International journal of biological sciencesRare 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 NeurophysiologyHummingbird sign in a patient with DNMT1-related disorder.
NeurocaseLenticulostriate vasculopathy in newborns: whole genome sequencing data analysis.
Frontiers in pediatricsCharacterization of novel and recurrent SPTLC2 variants in childhood-onset amyotrophic lateral sclerosis: Insights into sphingolipid dysregulation.
Journal of neuromuscular diseasesType IV Hereditary Sensory and Autonomic Neuropathy: Challenges and Functional Outcomes.
Journal of pediatric orthopedicsCongenital Insensitivity to Pain With Anhidrosis: First Reported Case in Nepal.
Clinical case reportsAdvances in the treatment of familial dysautonomia: what does the future hold?
Expert review of neurotherapeuticsDeciphering DST-associated disorders: biallelic variants affecting DST-b cause a congenital myopathy.
Brain : a journal of neurologySCN9A should not be considered an epilepsy gene; Refuting a gene-disease association.
Epilepsia[Peripheral Nerve: Cause and Pathology of Inherited Peripheral Neuropathy].
Brain and nerve = Shinkei kenkyu no shinpoRecurrent Osteomyelitis in a Paediatric Patient with a Novel NTRK1 Mutation: A Case Report on Congenital Insensitivity to Pain with Anhidrosis.
Children (Basel, Switzerland)Effect of SPTLC1 on type 2 diabetes mellitus.
World journal of diabetesFirst instance of pain in congenital pain insensitivity with anhidrosis.
Clinical neurology and neurosurgeryBlended phenotype of TECPR2-associated hereditary sensory-autonomic neuropathy and Temple syndrome.
Annals of clinical and translational neurologyA 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 reportsA novel mutation in the WNK1/HSN2 gene causing hereditary sensory and autonomic neuropathy type 2 in Chinese patient.
Journal of human geneticsGenetic and functional analyses of SPTLC1 in juvenile amyotrophic lateral sclerosis.
Journal of neurologyNovel TECPR2 variant in two cases of hereditary sensory and autonomic neuropathy type 9: insights from genetic characterization and comprehensive literature review.
BMC neurologyAtypical Presentation of Congenital Insensitivity to Pain With Anhidrosis Leading to Diagnostic Odyssey.
Molecular genetics & genomic medicineA Novel Pathogenic Mutation in WNK1 Gene Causing HSAN Type II in Three Siblings.
Journal of molecular neuroscience : MNCongenital Insensitivity to Pain With Anhidrosis Is Associated With Harlequin Color Change: A Survey Study.
Pediatric dermatologyA RETREG1 variant is associated with hereditary sensory and autonomic neuropathy with acral self-mutilation in purebred German Spitz.
Animal geneticsHereditary Sensory and Autonomic Neuropathy-Report of Two Cases in Siblings and Review of Literature.
Indian journal of dermatologyManagement Targeted Genetic Evaluation of an Idiopathic Neuropathy Cohort Through ATTRv Amyloidosis Screening.
HCA healthcare journal of medicineDevelopment of a Multiplexed Sphingolipids Method for Diagnosis of Inborn Errors of Ceramide Metabolism.
Clinical chemistrySensory-motor circuit is a therapeutic target for dystonia musculorum mice, a model of hereditary sensory and autonomic neuropathy 6.
Science advancesFam134c and Fam134b shape axonal endoplasmic reticulum architecture in vivo.
EMBO reportsFunctional and Molecular Characterization of New SPTLC1 Missense Variants in Patients with Hereditary Sensory and Autonomic Neuropathy Type 1 (HSAN1).
GenesA novel NTRK1 splice site variant causing congenital insensitivity to pain with anhidrosis in a Chinese family.
Frontiers in geneticsSerine Palmitoyltransferase (SPT)-related Neurodegenerative and Neurodevelopmental Disorders.
Journal of neuromuscular diseasesIdentification of a Novel Homozygous Mutation in PRDM12 Gene in a Patient with Hereditary Sensory and Autonomic Neuropathy Type VIII.
Archives of Iranian medicineA rare case of congenital insensitivity to pain with anhidrosis.
Paediatrics and international child healthClinical 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[Hereditary sensory and autonomic neuropathy 1E showing hyperreflexia: a case report].
Rinsho shinkeigaku = Clinical neurologyTECPR2-related hereditary sensory and autonomic neuropathy in two siblings from Palestine.
American journal of medical genetics. Part ADNMT1 Y495C mutation interferes with maintenance methylation of imprinting control regions.
The international journal of biochemistry & cell biologyPhenotypes of a toddler with hereditary sensory and autonomic neuropathy type IV: comparing with normal: A case report.
MedicineExploring CNS Involvement in Pain Insensitivity in Hereditary Sensory and Autonomic Neuropathy Type 4: Insights from Tc-99m ECD SPECT Imaging.
Tomography (Ann Arbor, Mich.)Congenital Insensitivity to Pain with Anhidrosis: A Case Report.
CureusMfsd7b facilitates choline transport and missense mutations affect choline transport function.
Cellular and molecular life sciences : CMLSRecurrent de novo SPTLC2 variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesis.
Journal of neurology, neurosurgery, and psychiatryExpression 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 neurologyMultisystemic RFC1-Related Disorder: Expanding the Phenotype Beyond Cerebellar Ataxia, Neuropathy, and Vestibular Areflexia Syndrome.
Neurology. Clinical practiceA novel HSPB1S139F mouse model of Charcot-Marie-Tooth Disease.
Prostaglandins & other lipid mediatorsRoles of dystonin isoforms in the maintenance of neural, muscle, and cutaneous tissues.
Anatomical science internationalAutonomic failure: Clinicopathologic, physiologic, and genetic aspects.
Handbook of clinical neurologyDST variants are responsible for neurogenic arthrogryposis multiplex congenita enlarging the spectrum of type VI hereditary sensory autonomic neuropathy.
Clinical geneticsOrthopedic Manifestations of Hereditary Sensory and Autonomic Neuropathy IV in a 10-Year-Old Patient.
The Iowa orthopaedic journalSPTLC1 p.Leu38Arg, a novel mutation associated with childhood ALS.
Biochimica et biophysica acta. Molecular and cell biology of lipidsMolecular characterization of wild-type and HSAN2B-linked FAM134B.
Molecular biology reportsExpanding the Knowledge of KIF1A-Dependent Disorders to a Group of Polish Patients.
GenesGenetic predisposition to ocular surface disorders and opportunities for gene-based therapies.
The ocular surfaceSpecific Deoxyceramide Species Correlate with Expression of Macular Telangiectasia Type 2 (MacTel2) in a SPTLC2 Carrier HSAN1 Family.
GenesSensorimotor control in the congenital absence of functional muscle spindles.
Experimental physiologyThe 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.
BiologySpectrum 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 NeurophysiologyA rare case of hereditary sensory and autonomic neuropathy type II.
Clinical case reportsSpatial proteomics reveals secretory pathway disturbances caused by neuropathy-associated TECPR2.
Nature communicationsNovel 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 NeurophysiologyIsolation and transfection of myenteric neurons from mice for patch-clamp applications.
Frontiers in molecular neuroscienceNGF and BDNF in pediatrics syndromes.
Neuroscience and biobehavioral reviewsHuman TrkAR649W mutation impairs nociception, sweating and cognitive abilities: a mouse model of HSAN IV.
Human molecular geneticsCongenital Insensitivity to Pain With Anhidrosis: A Case Report and Review of the Pertinent Literature.
CureusImpact of the coronavirus pandemic on families of patients with congenital insensitivity to pain with anhidrosis.
Pediatrics international : official journal of the Japan Pediatric SocietyCough as a neurological sign: What a clinician should know.
World journal of critical care medicineIdentification of sensory dysfunction and nervous structure changes in Fam134b knockout mice.
Neurological researchAssociation of variants in the KIF1A gene with amyotrophic lateral sclerosis.
Translational neurodegenerationKIF1A 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 neuroscienceA 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 degenerationAnesthetic management of a child with congenital insensitivity to pain with anhidrosis: A case report.
Frontiers in surgeryWNK1/HSN2 mediates neurite outgrowth and differentiation via a OSR1/GSK3β-LHX8 pathway.
Scientific reportsMulti-type RFC1 repeat expansions as the most common cause of hereditary sensory and autonomic neuropathy.
Frontiers in neurologyIsoform-specific mutation in Dystonin-b gene causes late-onset protein aggregate myopathy and cardiomyopathy.
eLifeHereditary Sensory and Autonomic Neuropathy Type II in a Female Child with Multiple Orthopaedic Ailments: Diagnosis and Operative Management.
Indian journal of orthopaedicsSPTLC1 variants associated with ALS produce distinct sphingolipid signatures through impaired interaction with ORMDL proteins.
The Journal of clinical investigationKeratoconus associated with hereditary sensory and autonomic neuropathy II.
Indian journal of ophthalmologyA case report: Anesthetic management for open-heart surgery in a child with congenital insensitivity to pain with anhidrosis.
Paediatric anaesthesiaNTRK1-related Hereditary Sensory and Autonomic Neuropathy Type 4: The Role of the Histamine Challenge Test.
Child neurology openGenetic pain loss disorders.
Nature reviews. Disease primersNew Insights into the Neuromyogenic Spectrum of a Gain of Function Mutation in SPTLC1.
GenesNovel RETREG1 (FAM134B) founder allele is linked to HSAN2B and renal disease in a Turkish family.
American journal of medical genetics. Part AHereditary Sensory and Autonomic Neuropathy Type VIII: Congenital Insensitivity to Pain with Anhidrosis.
Indian dermatology online journalPathogenic DST sequence variants result in either epidermolysis bullosa simplex (EBS) or hereditary sensory and autonomic neuropathy type 6 (HSAN-VI).
Experimental dermatologyHereditary Sensory and Autonomic Neuropathy: A Case Series of Six Children.
Neurology IndiaHereditary Autonomic Neuropathy of the Oral Cavity and its Management.
Iranian journal of child neurologyPregnancy in hereditary sensory and autonomic neuropathy type V: A case report and literature review.
Taiwanese journal of obstetrics & gynecologyA neuropathy-associated kinesin KIF1A mutation hyper-stabilizes the motor-neck interaction during the ATPase cycle.
The EMBO journalA 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 ADevelopmental regulation of neuronal gene expression by Elongator complex protein 1 dosage.
Journal of genetics and genomics = Yi chuan xue baoPrecision mouse models of Yars/dominant intermediate Charcot-Marie-Tooth disease type C and Sptlc1/hereditary sensory and autonomic neuropathy type 1.
Journal of anatomy1-Deoxysphinganine initiates adaptive responses to serine and glycine starvation in cancer cells via proteolysis of sphingosine kinase.
Journal of lipid researchA 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 reportsGeneration of a transgenic mouse embryonic stem cell line expressing Dnmt1Y495C mutation associated with HSAN1E disorder.
Stem cell researchNot Another Case Of Juvenile Idiopathic Arthritis: Congenital Insensitivity To Pain Presenting With Joint Problems.
Journal of Ayub Medical College, Abbottabad : JAMCHereditary sensory and autonomic neuropathy in a family of mixed breed dogs associated with a novel RETREG1 variant.
Journal of veterinary internal medicineTowards personalized medicine for amyotrophic lateral sclerosis.
Trends in endocrinology and metabolism: TEMGenotype and phenotype distribution of 435 patients with Charcot-Marie-Tooth disease from central south China.
European journal of neurologyChildhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis.
Nature medicineUnderstanding pain perception through genetic painlessness diseases: The role of NGF and proNGF.
Pharmacological researchLate-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 leprologyThe 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. BiomembranesExpanding the Genotypic Spectrum of Congenital Sensory and Autonomic Neuropathies Using Whole-Exome Sequencing.
Neurology. GeneticsClinical, neuroimaging, and molecular spectrum of TECPR2-associated hereditary sensory and autonomic neuropathy with intellectual disability.
Human mutationAAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
Trends in molecular medicineCerebellar Ataxia as a Common Clinical Presentation Associated with DNMT1 p.Y511H and a Review of the Literature.
Journal of molecular neuroscience : MN[A case report of hereditary sensory and autonomic neuropathy type Ⅶ].
Zhonghua er ke za zhi = Chinese journal of pediatricsReduced Myocardial Uptake of 123I-MIBG in Congenital Insensitivity to Pain With Anhidrosis.
Clinical nuclear medicineFrequency and burden of gastrointestinal symptoms in familial dysautonomia.
Clinical autonomic research : official journal of the Clinical Autonomic Research SocietyBilateral harlequin syndrome, unilateral Horner syndrome, and Riga-Fede disease as presenting features of hereditary sensory and autonomic neuropathy type IV.
Pediatric dermatologyHigh prevalence of carpal tunnel syndrome in individuals with rare nerve growth factor-beta mutation.
Brain communications1-Deoxysphingolipids cause autophagosome and lysosome accumulation and trigger NLRP3 inflammasome activation.
AutophagySisters 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 dermatologyHeterozygous KIF1A variants underlie a wide spectrum of neurodevelopmental and neurodegenerative disorders.
Journal of medical geneticsDiverse dystonin gene mutations cause distinct patterns of Dst isoform deficiency and phenotypic heterogeneity in Dystonia musculorum mice.
Disease models & mechanismsElbow proprioception is normal in patients with a congenital absence of functional muscle spindles.
The Journal of physiologyDisruption of dystonin in Schwann cells results in late-onset neuropathy and sensory ataxia.
GliaCanonical and 1-Deoxy(methyl) Sphingoid Bases: Tackling the Effect of the Lipid Structure on Membrane Biophysical Properties.
Langmuir : the ACS journal of surfaces and colloidsRetrograde nerve growth factor signaling abnormalities in familial dysautonomia.
The Journal of clinical investigationA Rare KIF1A Missense Mutation Enhances Synaptic Function and Increases Seizure Activity.
Frontiers in geneticsPopulation Study of Hand and Wrist Manifestations of Congenital Insensitivity to Pain.
Hand (New York, N.Y.)A novel biallelic single base insertion in WNK1 in a Pakistani family with congenital insensitivity to pain.
Journal of human geneticsHSAN-VI: A spectrum disorder based on dystonin isoform expression.
Neurology. GeneticsCataplexy 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 MedicineFAM134B oligomerization drives endoplasmic reticulum membrane scission for ER-phagy.
The EMBO journalCharacterization of gastrointestinal pathologies in the dystonia musculorum mouse model for hereditary sensory and autonomic neuropathy type VI.
Neurogastroenterology and motilityFirst 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 NeurophysiologyHereditary Sensory and Autonomic Neuropathy 2B Caused by a Novel RETREG1 Mutation (c.765dupT) and Paternal Uniparental Isodisomy of Chromosome 5.
Frontiers in geneticsThe 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 NeuroscienceFocal Epithelial Hyperplasia in a Child with Hereditary Sensory and Autonomic Neuropathy type IV: A Rare Co-Occurrence.
Journal of dentistry for children (Chicago, Ill.)Serine and Lipid Metabolism in Macular Disease and Peripheral Neuropathy.
The New England journal of medicineCytotoxicity of 1-deoxysphingolipid unraveled by genome-wide genetic screens and lipidomics in Saccharomyces cerevisiae.
Molecular biology of the cellDe novo pathogenic DNM1L variant in a patient diagnosed with atypical hereditary sensory and autonomic neuropathy.
Molecular genetics & genomic medicineCongenital Insensitivity to Pain with Anhidrosis: A Case with Self-Inflicted Oral Ulcerations.
Journal of dentistry for children (Chicago, Ill.)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 NeuroscienceThe coexistence of a novel WNK1 variant and a copy number variation causes hereditary sensory and autonomic neuropathy type IIA.
BMC medical geneticsA Novel Variant (Asn177Asp) in SPTLC2 Causing Hereditary Sensory Autonomic Neuropathy Type 1C.
Neuromolecular medicineDNMT1-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 NeurophysiologyChemoreflex failure and sleep-disordered breathing in familial dysautonomia: Implications for sudden death during sleep.
Autonomic neuroscience : basic & clinicalHereditary sensory and autonomic neuropathy type IC accompanied by upper motor neuron abnormalities and type II juxtafoveal retinal telangiectasias.
Journal of the peripheral nervous system : JPNSExtreme Ends of Pain Sensitivity in SCN9A Mutation Variants: Case Report and Literature Review.
Innovations in clinical neuroscienceATL3 Is a Tubular ER-Phagy Receptor for GABARAP-Mediated Selective Autophagy.
Current biology : CBHeterotopic ossifications and Charcot joints: Congenital insensitivity to pain with anhidrosis (CIPA) and a novel NTRK1 gene mutation.
European journal of medical geneticsUncoupling neurotrophic function from nociception of nerve growth factor: what can be learned from a rare human disease?
Neural regeneration researchRandomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1.
NeurologyPlantar Ulcers and Neuropathic Arthropathies: Associated Diseases, Polyneuropathy Correlates, and Risk Covariates.
Advances in skin & wound careCholinergic striatal neurons are increased in HSAN V homozygous mice despite reduced NGF bioavailability.
Biochemical and biophysical research communicationsComprehensive Computational Analysis of Protein Phenotype Changes Due to Plausible Deleterious Variants of Human SPTLC1 Gene.
International journal of molecular and cellular medicineThe novel de novo mutation of KIF1A gene as the cause for Spastic paraplegia 30 in a Japanese case.
eNeurologicalSciA novel nonsense mutation in WNK1/HSN2 associated with sensory neuropathy and limb destruction in four siblings of a large Iranian pedigree.
BMC neurologyV144D Mutation of SPTLC1 Can Present with Both Painful and Painless Phenotypes in Hereditary Sensory and Autonomic Neuropathies Type I.
Case reports in geneticsPainless Nerve Growth Factor: A TrkA biased agonist mediating a broad neuroprotection via its actions on microglia cells.
Pharmacological researchGeneration 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 researchMolecular diagnosis of inherited peripheral neuropathies by targeted next-generation sequencing: molecular spectrum delineation.
BMJ openIdentification of a novel DNMT1 mutation in a Chinese patient with hereditary sensory and autonomic neuropathy type IE.
BMC neurologySensory neuropathy-causing mutations in ATL3 affect ER-mitochondria contact sites and impair axonal mitochondrial distribution.
Human molecular geneticsResting Energy Expenditure in Patients With Familial Dysautonomia: A Preliminary Study.
Journal of pediatric gastroenterology and nutritionA third HSAN5 mutation disrupts the nerve growth factor furin cleavage site.
Molecular painOffice-Based Anesthetic and Oral Surgical Management of a Child With Hereditary Sensory Autonomic Neuropathy Type IV: A Case Report.
Anesthesia progressImpaired sensorimotor control of the hand in congenital absence of functional muscle spindles.
Journal of neurophysiologyIdentification of a novel mutation of the NTRK1 gene in patients with congenital insensitivity to pain with anhidrosis (CIPA).
GeneHereditary sensory and autonomic neuropathy in a male child: 'The other side of not feeling pain'.
BMJ case reportsTsc3 regulates SPT amino acid choice in Saccharomyces cerevisiae by promoting alanine in the sphingolipid pathway.
Journal of lipid researchA Child Presenting with Recurrent Corneal Ulcers: Hereditary Sensory and Autonomic Neuropathy IV (HSAN IV).
Neuro-ophthalmology (Aeolus Press)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 SurgeonsLate-onset hereditary sensory and autonomic neuropathy expands the phenotypic spectrum of MFN2-related diseases.
Neuropathology : official journal of the Japanese Society of NeuropathologyDystonin-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 geneticsMidface 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 dermatologySensory-Neuropathy-Causing Mutations in ATL3 Cause Aberrant ER Membrane Tethering.
Cell reportsRepeated hyperhidrosis and chilblain-like swelling with ulceration of the fingers and toes in hereditary sensory and autonomic neuropathy type II.
The Journal of dermatologyCerebellar ataxia, neuropathy, vestibular areflexia syndrome (CANVAS) with chronic cough and preserved muscle stretch reflexes: evidence for selective sparing of afferent Ia fibres.
Journal of neurologyHereditary Sensory and Autonomic Neuropathy Presenting With Mutilating Trophic Ulcers.
Wounds : a compendium of clinical research and practiceGlycogen synthase kinase 3ß functions as a positive effector in the WNK signaling pathway.
PloS oneSwedish 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 NeuroscienceFounder mutation in IKBKAP gene causes vestibular impairment in familial dysautonomia.
Clinical neurophysiology : official journal of the International Federation of Clinical NeurophysiologyRETREG1 (FAM134B): A new player in human diseases: 15 years after the discovery in cancer.
Journal of cellular physiology[A case of hereditary sensory and autonomic neuropathy type 1E with frontal lobe dysfunction as an initial symptom].
Rinsho shinkeigaku = Clinical neurologyAnesthetic Management of a Patient With De Novo Hereditary Sensory and Autonomic Neuropathy, Type VII: A Case Report.
A&A practiceMotoneuron degeneration in the trigeminal motor nucleus innervating the masseter muscle in Dystonia musculorum mice.
Neurochemistry internationalClinical and metabolic consequences of L-serine supplementation in hereditary sensory and autonomic neuropathy type 1C.
Cold Spring Harbor molecular case studiesRiley-Day Syndrome in a Hispanic Infant of Non-Jewish Ashkenazi Descent.
Journal of clinical and diagnostic research : JCDROral 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 reportsBPAG1 in muscles: Structure and function in skeletal, cardiac and smooth muscle.
Seminars in cell & developmental biologyAnimal and cellular models of familial dysautonomia.
Clinical autonomic research : official journal of the Clinical Autonomic Research Society[ORO-DENTO-FACIAL MANIFESTATIONS IN PATIENTS WITH FAMILIAL DYSAUTONOMIA].
HarefuahSudden Unexpected Death During Sleep in Familial Dysautonomia: A Case-Control Study.
SleepBGP-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 AmericaWNK1/HSN2 founder mutation in patients with hereditary sensory and autonomic neuropathy: A Japanese cohort study.
Clinical geneticsIdentification 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 researchExome sequencing identifies novel NTRK1 mutations in patients with HSAN-IV phenotype.
American journal of medical genetics. Part AAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
Ainda não temos associações cadastradas para Neuropatia sensitiva e autonômica hereditária.
É de uma associação que acompanha esta doença? Fale com a gente →
Doença com base genética
Um médico geneticista pode ajudar no diagnóstico de Neuropatia sensitiva e autonômica hereditária e no aconselhamento genético da família.
Doenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
Perguntas frequentes
O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu
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.
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
Neuropatia sensitiva e autonômica hereditária
📋 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
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Indexação biomédica
- fonte: MeSH (NLM)
- Dado público estruturado
- fonte: Wikidata
- Ensaios clínicos
- fonte: ClinicalTrials.gov