Raras
Buscar doenças, sintomas, genes...
Doença de Parkinson de início juvenil
ORPHA:2828CID-10 · G20CID-11 · 8A00.00DOENÇA RARA

Uma forma da doença de Parkinson (DP) que se manifesta entre os 21 e 45 anos de idade, caracterizada por rigidez, cãibras dolorosas seguidas de tremor, lentidão dos movimentos, espasmos musculares involuntários, dificuldades para andar e quedas, além de outros sintomas não relacionados ao movimento. A doença geralmente avança de forma lenta e, na maioria dos casos, há uma resposta mais acentuada ao tratamento com medicamentos que agem na dopamina.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Uma forma da doença de Parkinson (DP) que se manifesta entre os 21 e 45 anos de idade, caracterizada por rigidez, cãibras dolorosas seguidas de tremor, lentidão dos movimentos, espasmos musculares involuntários, dificuldades para andar e quedas, além de outros sintomas não relacionados ao movimento. A doença geralmente avança de forma lenta e, na maioria dos casos, há uma resposta mais acentuada ao tratamento com medicamentos que agem na dopamina.

Pesquisas ativas
4 ensaios
15 total registrados no ClinicalTrials.gov
Publicações científicas
25 artigos
Último publicado: 2026 Jun

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-5 / 10 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
15.0
Europe
Início
Adult
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: G20
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (2)
0202010694
Sequenciamento completo do exoma (WES)genetic_test
0301070040
Atendimento em reabilitação — doenças rarasrehabilitation
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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

🧠
Neurológico
31 sintomas
💪
Músculos
11 sintomas
🫃
Digestivo
6 sintomas
👁️
Olhos
4 sintomas
🦴
Ossos e articulações
4 sintomas
👂
Ouvidos
2 sintomas

+ 58 sintomas em outras categorias

Características mais comuns

90%prev.
Rigidez
Muito frequente (99-80%)
55%prev.
Apatia
Frequente (79-30%)
55%prev.
Depressão
Frequente (79-30%)
55%prev.
Discinesia
Frequente (79-30%)
55%prev.
Tremor
Frequente (79-30%)
55%prev.
Alucinações
Frequente (79-30%)
121sintomas
Muito frequente (1)
Frequente (6)
Ocasional (23)
Muito raro (4)
Sem dados (87)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 121 características clínicas mais associadas, ordenadas por frequência.

RigidezRigidity
Muito frequente (99-80%)90%
ApatiaApathy
Frequente (79-30%)55%
DepressãoDepression
Frequente (79-30%)55%
DiscinesiaDyskinesia
Frequente (79-30%)55%
Tremor
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico25PubMed
Últimos 10 anos13publicações
Pico20234 papers
Linha do tempo
2026Hoje · 2026🧪 2004Primeiro ensaio clínico📈 2023Ano 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

12 genes identificados com associação a esta condição. Padrão de herança: Autosomal recessive.

PINK1Serine/threonine-protein kinase PINK1, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Serine/threonine-protein kinase which acts as a sensor of mitochondrial damage and protects against mitochondrial dysfunction during cellular stress (PubMed:40080546). It phosphorylates mitochondrial proteins to coordinate mitochondrial quality control mechanisms that remove and replace dysfunctional mitochondrial components (PubMed:14607334, PubMed:15087508, PubMed:18443288, PubMed:18957282, PubMed:19229105, PubMed:19966284, PubMed:20404107, PubMed:20547144, PubMed:20798600, PubMed:22396657, Pu

LOCALIZAÇÃO

Mitochondrion outer membraneMitochondrion inner membraneCytoplasm, cytosol

VIAS BIOLÓGICAS (2)
PINK1-PRKN Mediated MitophagyFOXO-mediated transcription of cell death genes
MECANISMO DE DOENÇA

Parkinson disease 6

An early-onset form of Parkinson disease, a neurodegenerative disorder characterized by parkinsonian signs such as rigidity, resting tremor and bradykinesia. A subset of patients manifest additional symptoms including hyperreflexia, autonomic instability, dementia and psychiatric disturbances. Symptoms show diurnal fluctuation and can improve after sleep. PARK6 pathogenesis involves respiratory complex I deficiency causing mitochondrial depolarization and dysfunction. Inheritance is autosomal recessive.

EXPRESSÃO TECIDUAL(Ubíquo)
Músculo esquelético
84.3 TPM
Brain Frontal Cortex BA9
68.5 TPM
Testículo
64.4 TPM
Glândula adrenal
61.7 TPM
Córtex cerebral
61.0 TPM
OUTRAS DOENÇAS (2)
autosomal recessive early-onset Parkinson disease 6young-onset Parkinson disease
HGNC:14581UniProt:Q9BXM7
ATP13A2Polyamine-transporting ATPase 13A2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

ATPase which acts as a lysosomal polyamine exporter with high affinity for spermine (PubMed:31996848). Also stimulates cellular uptake of polyamines and protects against polyamine toxicity (PubMed:31996848). Plays a role in intracellular cation homeostasis and the maintenance of neuronal integrity (PubMed:22186024). Contributes to cellular zinc homeostasis (PubMed:24603074). Confers cellular protection against Mn(2+) and Zn(2+) toxicity and mitochondrial stress (PubMed:26134396). Required for pr

LOCALIZAÇÃO

Lysosome membraneLate endosome membraneEndosome, multivesicular body membraneCytoplasmic vesicle, autophagosome membrane

VIAS BIOLÓGICAS (1)
Ion transport by P-type ATPases
MECANISMO DE DOENÇA

Kufor-Rakeb syndrome

A rare form of autosomal recessive juvenile or early-onset, levodopa-responsive parkinsonism. In addition to typical parkinsonian signs, clinical manifestations of Kufor-Rakeb syndrome include behavioral problems, facial tremor, pyramidal tract dysfunction, supranuclear gaze palsy, and dementia.

OUTRAS DOENÇAS (3)
Kufor-Rakeb syndromeautosomal recessive spastic paraplegia type 78parkinsonism due to ATP13A2 deficiency
HGNC:30213UniProt:Q9NQ11
PODXLPodocalyxinDisease-causing germline mutation(s) (loss of function) inRestrito
FUNÇÃO

Involved in the regulation of both adhesion and cell morphology and cancer progression. Functions as an anti-adhesive molecule that maintains an open filtration pathway between neighboring foot processes in the podocyte by charge repulsion. Acts as a pro-adhesive molecule, enhancing the adherence of cells to immobilized ligands, increasing the rate of migration and cell-cell contacts in an integrin-dependent manner. Induces the formation of apical actin-dependent microvilli. Involved in the form

LOCALIZAÇÃO

Apical cell membraneCell projection, lamellipodiumCell projection, filopodiumCell projection, ruffleCell projection, microvillusMembrane raftMembrane

VIAS BIOLÓGICAS (1)
Cytosolic sulfonation of small molecules
EXPRESSÃO TECIDUAL(Ubíquo)
Rim - Córtex
161.5 TPM
Pulmão
133.5 TPM
Tireoide
89.0 TPM
Útero
85.2 TPM
Fallopian Tube
74.8 TPM
OUTRAS DOENÇAS (2)
atypical juvenile parkinsonismyoung-onset Parkinson disease
HGNC:9171UniProt:O00592
LRRK2Leucine-rich repeat serine/threonine-protein kinase 2Candidate gene tested inTolerante
FUNÇÃO

Serine/threonine-protein kinase which phosphorylates a broad range of proteins involved in multiple processes such as neuronal plasticity, innate immunity, autophagy, and vesicle trafficking (PubMed:17114044, PubMed:20949042, PubMed:21850687, PubMed:22012985, PubMed:23395371, PubMed:24687852, PubMed:25201882, PubMed:26014385, PubMed:26824392, PubMed:27830463, PubMed:28720718, PubMed:29125462, PubMed:29127255, PubMed:29212815, PubMed:30398148, PubMed:30635421). Is a key regulator of RAB GTPases b

LOCALIZAÇÃO

Cytoplasmic vesiclePerikaryonGolgi apparatus membraneCell projection, axonCell projection, dendriteEndoplasmic reticulum membraneCytoplasmic vesicle, secretory vesicle, synaptic vesicle membraneEndosomeLysosomeMitochondrion outer membraneCytoplasm, cytoskeletonCytoplasmic vesicle, phagosome

VIAS BIOLÓGICAS (1)
PTK6 promotes HIF1A stabilization
MECANISMO DE DOENÇA

Parkinson disease 8

A slowly progressive neurodegenerative disorder characterized by bradykinesia, rigidity, resting tremor, postural instability, neuronal loss in the substantia nigra, and the presence of neurofibrillary MAPT (tau)-positive and Lewy bodies in some patients.

EXPRESSÃO TECIDUAL(Ubíquo)
Pulmão
29.7 TPM
Sangue
21.6 TPM
Nervo tibial
17.4 TPM
Aorta
12.6 TPM
Artéria tibial
11.9 TPM
OUTRAS DOENÇAS (3)
late-onset Parkinson diseaseyoung-onset Parkinson diseaseautosomal dominant Parkinson disease 8
HGNC:18618UniProt:Q5S007
PARK7Parkinson disease protein 7Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Multifunctional protein with controversial molecular function which plays an important role in cell protection against oxidative stress and cell death acting as oxidative stress sensor and redox-sensitive chaperone and protease (PubMed:12796482, PubMed:17015834, PubMed:18711745, PubMed:19229105, PubMed:20304780, PubMed:25416785, PubMed:26995087, PubMed:28993701). It is involved in neuroprotective mechanisms like the stabilization of NFE2L2 and PINK1 proteins, male fertility as a positive regulat

LOCALIZAÇÃO

Cell membraneCytoplasmNucleusMembrane raftMitochondrionEndoplasmic reticulum

VIAS BIOLÓGICAS (1)
SUMOylation of transcription cofactors
MECANISMO DE DOENÇA

Parkinson disease 7

A neurodegenerative disorder characterized by resting tremor, postural tremor, bradykinesia, muscular rigidity, anxiety and psychotic episodes. PARK7 has onset before 40 years, slow progression and initial good response to levodopa. Some patients may show traits reminiscent of amyotrophic lateral sclerosis-parkinsonism/dementia complex (Guam disease).

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
303.7 TPM
Fibroblastos
302.9 TPM
Cérebro - Hemisfério cerebelar
277.7 TPM
Aorta
273.1 TPM
Nervo tibial
258.0 TPM
OUTRAS DOENÇAS (3)
autosomal recessive early-onset Parkinson disease 7amyotrophic lateral sclerosis-parkinsonism-dementia complexyoung-onset Parkinson disease
HGNC:16369UniProt:Q99497
VPS13CIntermembrane lipid transfer protein VPS13CDisease-causing germline mutation(s) (loss of function) inTolerante
FUNÇÃO

Mediates the transfer of lipids between membranes at organelle contact sites (By similarity). Necessary for proper mitochondrial function and maintenance of mitochondrial transmembrane potential (PubMed:26942284). Involved in the regulation of PINK1/PRKN-mediated mitophagy in response to mitochondrial depolarization (PubMed:26942284)

LOCALIZAÇÃO

Mitochondrion outer membraneLipid dropletEndoplasmic reticulum membraneLysosome membraneLate endosome membrane

MECANISMO DE DOENÇA

Parkinson disease 23, autosomal recessive, early onset

An autosomal recessive, early-onset form of Parkinson disease, a complex neurodegenerative disorder characterized by bradykinesia, resting tremor, muscular rigidity and postural instability, as well as by a clinically significant response to treatment with levodopa. The pathology involves the loss of dopaminergic neurons in the substantia nigra and the presence of Lewy bodies (intraneuronal accumulations of aggregated proteins), in surviving neurons in various areas of the brain.

EXPRESSÃO TECIDUAL(Ubíquo)
Nervo tibial
32.4 TPM
Linfócitos
25.5 TPM
Pituitária
25.5 TPM
Ovário
24.2 TPM
Cerebelo
23.1 TPM
OUTRAS DOENÇAS (2)
autosomal recessive early-onset Parkinson disease 23young-onset Parkinson disease
HGNC:23594UniProt:Q709C8
DNAJC6AuxilinDisease-causing germline mutation(s) (loss of function) inAltamente restrito
FUNÇÃO

May act as a protein phosphatase and/or a lipid phosphatase. Co-chaperone that recruits HSPA8/HSC70 to clathrin-coated vesicles (CCVs) and promotes the ATP-dependent dissociation of clathrin from CCVs and participates in clathrin-mediated endocytosis of synaptic vesicles and their recycling and also in intracellular trafficking (PubMed:18489706). Firstly, binds tightly to the clathrin cages, at a ratio of one DNAJC6 per clathrin triskelion. The HSPA8:ATP complex then binds to the clathrin-auxili

LOCALIZAÇÃO

Cytoplasmic vesicle, clathrin-coated vesicle

VIAS BIOLÓGICAS (3)
Clathrin-mediated endocytosisLysosome Vesicle BiogenesisGolgi Associated Vesicle Biogenesis
MECANISMO DE DOENÇA

Parkinson disease 19A, juvenile-onset

A juvenile form of Parkinson disease, a complex neurodegenerative disorder characterized by bradykinesia, resting tremor, muscular rigidity and postural instability, as well as by a clinically significant response to treatment with levodopa. The pathology involves the loss of dopaminergic neurons in the substantia nigra and the presence of Lewy bodies (intraneuronal accumulations of aggregated proteins), in surviving neurons in various areas of the brain. PARK19A is characterized by onset of parkinsonian symptoms in the first or second decade of life. Some patients may have additional neurologic features, including intellectual disability and seizures.

EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
80.0 TPM
Brain Frontal Cortex BA9
69.8 TPM
Brain Spinal cord cervical c-1
62.9 TPM
Cerebelo
51.3 TPM
Hipotálamo
49.0 TPM
OUTRAS DOENÇAS (3)
juvenile onset Parkinson disease 19Aatypical juvenile parkinsonismyoung-onset Parkinson disease
HGNC:15469UniProt:O75061
HTRA2Serine protease HTRA2, mitochondrialCandidate gene tested inTolerante
FUNÇÃO

Serine protease that shows proteolytic activity against a non-specific substrate beta-casein (PubMed:10873535). Promotes apoptosis by either relieving the inhibition of BIRC proteins on caspases, leading to an increase in caspase activity; or by a BIRC inhibition-independent, caspase-independent and serine protease activity-dependent mechanism (PubMed:15200957). Cleaves BIRC6 and relieves its inhibition on CASP3, CASP7 and CASP9, but it is also prone to inhibition by BIRC6 (PubMed:36758104, PubM

LOCALIZAÇÃO

Mitochondrion intermembrane spaceMitochondrion membraneEndoplasmic reticulum

VIAS BIOLÓGICAS (2)
Mitochondrial unfolded protein response (UPRmt)Mitochondrial protein degradation
MECANISMO DE DOENÇA

3-methylglutaconic aciduria 8

An autosomal recessive inborn error of metabolism resulting in early death. Clinical features include extreme hypertonia observed at birth, alternating with hypotonia, subsequent appearance of extrapyramidal symptoms, lack of psychomotor development, microcephaly, and intractable seizures. Patients show lactic acidemia, 3-methylglutaconic aciduria, intermittent neutropenia, and progressive brain atrophy.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
62.3 TPM
Útero
44.3 TPM
Cervix Ectocervix
44.1 TPM
Ovário
44.0 TPM
Cervix Endocervix
43.3 TPM
OUTRAS DOENÇAS (3)
3-methylglutaconic aciduria type 8young-onset Parkinson diseaseParkinson disease 13, autosomal dominant, susceptibility to
HGNC:14348UniProt:O43464
UCHL1Ubiquitin carboxyl-terminal hydrolase isozyme L1Candidate gene tested inAltamente restrito
FUNÇÃO

Deubiquitinase that plays a role in the regulation of several processes such as maintenance of synaptic function, cardiac function, inflammatory response or osteoclastogenesis (PubMed:22212137, PubMed:23359680). Abrogates the ubiquitination of multiple proteins including WWTR1/TAZ, EGFR, HIF1A and beta-site amyloid precursor protein cleaving enzyme 1/BACE1 (PubMed:22212137, PubMed:25615526). In addition, recognizes and hydrolyzes a peptide bond at the C-terminal glycine of ubiquitin to maintain

LOCALIZAÇÃO

CytoplasmEndoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
UCH proteinases
MECANISMO DE DOENÇA

Parkinson disease 5

A complex neurodegenerative disorder with manifestations ranging from typical Parkinson disease to dementia with Lewy bodies. Clinical features include parkinsonian symptoms (resting tremor, rigidity, postural instability and bradykinesia), dementia, diffuse Lewy body pathology, autonomic dysfunction, hallucinations and paranoia.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Ubíquo)
Brain Frontal Cortex BA9
1197.5 TPM
Córtex cerebral
948.1 TPM
Hipotálamo
894.5 TPM
Brain Anterior cingulate cortex BA24
700.8 TPM
Brain Nucleus accumbens basal ganglia
687.1 TPM
OUTRAS DOENÇAS (4)
early-onset progressive neurodegeneration-blindness-ataxia-spasticity syndromespastic paraplegia 79A, autosomal dominant, with ataxiayoung-onset Parkinson diseaseParkinson disease 5, autosomal dominant, susceptibility to
HGNC:12513UniProt:P09936
SNCAAlpha-synucleinCandidate gene tested inModerado
FUNÇÃO

Neuronal protein that plays several roles in synaptic activity such as regulation of synaptic vesicle trafficking and subsequent neurotransmitter release (PubMed:20798282, PubMed:26442590, PubMed:28288128, PubMed:30404828). Participates as a monomer in synaptic vesicle exocytosis by enhancing vesicle priming, fusion and dilation of exocytotic fusion pores (PubMed:28288128, PubMed:30404828). Mechanistically, acts by increasing local Ca(2+) release from microdomains which is essential for the enha

LOCALIZAÇÃO

CytoplasmMembraneNucleusSynapseSecretedCell projection, axon

VIAS BIOLÓGICAS (2)
PKR-mediated signalingAmyloid fiber formation
EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
106.1 TPM
Brain Frontal Cortex BA9
106.0 TPM
Brain Anterior cingulate cortex BA24
75.0 TPM
Cerebelo
73.9 TPM
Nervo tibial
67.0 TPM
OUTRAS DOENÇAS (6)
autosomal dominant Parkinson disease 4autosomal dominant Parkinson disease 1Lewy body dementiayoung-onset Parkinson disease
HGNC:11138UniProt:P37840
PRKNE3 ubiquitin-protein ligase parkinDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Functions within a multiprotein E3 ubiquitin ligase complex, catalyzing the covalent attachment of ubiquitin moieties onto substrate proteins (PubMed:10888878, PubMed:10973942, PubMed:11431533, PubMed:12150907, PubMed:12628165, PubMed:15105460, PubMed:16135753, PubMed:21376232, PubMed:21532592, PubMed:22396657, PubMed:23620051, PubMed:23754282, PubMed:24660806, PubMed:24751536, PubMed:29311685, PubMed:32047033). Substrates include SYT11 and VDAC1 (PubMed:29311685, PubMed:32047033). Other substra

LOCALIZAÇÃO

Cytoplasm, cytosolNucleusEndoplasmic reticulumMitochondrionMitochondrion outer membraneCell projection, neuron projectionPostsynaptic densityPresynapse

VIAS BIOLÓGICAS (5)
AggrephagyPINK1-PRKN Mediated MitophagyAntigen processing: Ubiquitination & Proteasome degradationAmyloid fiber formationJosephin domain DUBs
MECANISMO DE DOENÇA

Parkinson disease

A complex neurodegenerative disorder characterized by bradykinesia, resting tremor, muscular rigidity and postural instability. Additional features are characteristic postural abnormalities, dysautonomia, dystonic cramps, and dementia. The pathology of Parkinson disease involves the loss of dopaminergic neurons in the substantia nigra and the presence of Lewy bodies (intraneuronal accumulations of aggregated proteins), in surviving neurons in various areas of the brain. The disease is progressive and usually manifests after the age of 50 years, although early-onset cases (before 50 years) are known. The majority of the cases are sporadic suggesting a multifactorial etiology based on environmental and genetic factors. However, some patients present with a positive family history for the disease. Familial forms of the disease usually begin at earlier ages and are associated with atypical clinical features.

OUTRAS DOENÇAS (4)
ovarian cancerautosomal recessive juvenile Parkinson disease 2lung canceryoung-onset Parkinson disease
HGNC:8607UniProt:O60260
SYNJ1Synaptojanin-1Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Phosphatase that acts on various phosphoinositides, including phosphatidylinositol 4-phosphate, phosphatidylinositol (4,5)-bisphosphate and phosphatidylinositol (3,4,5)-trisphosphate (PubMed:23804563, PubMed:27435091). Has a role in clathrin-mediated endocytosis (By similarity). Hydrolyzes PIP2 bound to actin regulatory proteins resulting in the rearrangement of actin filaments downstream of tyrosine kinase and ASH/GRB2 (By similarity)

LOCALIZAÇÃO

Cytoplasm, perinuclear region

VIAS BIOLÓGICAS (4)
Synthesis of PIPs at the plasma membraneClathrin-mediated endocytosisSynthesis of IP3 and IP4 in the cytosolSynthesis of IP2, IP, and Ins in the cytosol
MECANISMO DE DOENÇA

Parkinson disease 20, early-onset

An early-onset form of Parkinson disease, a complex neurodegenerative disorder characterized by bradykinesia, resting tremor, muscular rigidity and postural instability, as well as by a clinically significant response to treatment with levodopa. The pathology involves the loss of dopaminergic neurons in the substantia nigra and the presence of Lewy bodies (intraneuronal accumulations of aggregated proteins), in surviving neurons in various areas of the brain. PARK20 is characterized by young adult-onset of parkinsonism. Additional features may include seizures, cognitive decline, abnormal eye movements, and dystonia.

EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
38.4 TPM
Brain Frontal Cortex BA9
35.2 TPM
Cerebelo
33.2 TPM
Córtex cerebral
21.4 TPM
Testículo
18.7 TPM
OUTRAS DOENÇAS (5)
early-onset Parkinson disease 20developmental and epileptic encephalopathy, 53atypical juvenile parkinsonismundetermined early-onset epileptic encephalopathy
HGNC:11503UniProt:O43426

Variantes genéticas (ClinVar)

319 variantes patogênicas registradas no ClinVar.

🧬 PINK1: NM_032409.3(PINK1):c.131del (p.Glu44fs) ()
🧬 PINK1: NM_032409.3(PINK1):c.619C>T (p.Arg207Ter) ()
🧬 PINK1: NM_032409.3(PINK1):c.1124-1G>A ()
🧬 PINK1: NM_032409.3(PINK1):c.1080_1081del (p.His360fs) ()
🧬 PINK1: NM_032409.3(PINK1):c.378G>T (p.Gln126His) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 33 variantes classificadas pelo ClinVar.

28
5
Patogênica (84.8%)
VUS (15.2%)
VARIANTES MAIS SIGNIFICATIVAS
VPS13C: NM_020821.3(VPS13C):c.1119G>A (p.Trp373Ter) [Likely pathogenic]
VPS13C: NM_020821.3(VPS13C):c.5698C>T (p.Gln1900Ter) [Likely pathogenic]
VPS13C: NM_020821.3(VPS13C):c.5462T>A (p.Leu1821Ter) [Likely pathogenic]
VPS13C: NM_020821.3(VPS13C):c.3331G>T (p.Gly1111Ter) [Likely pathogenic]
VPS13C: NM_020821.3(VPS13C):c.2408+1G>A [Likely pathogenic]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

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.
3Fase 31
1Fase 12
·Pré-clínico7
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 10 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Doença de Parkinson de início juvenil

🗺️

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

🟢 Recrutando agora

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

Outros ensaios clínicos

15 ensaios clínicos encontrados, 4 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

Timeline de publicações
15 papers (10 anos)
#1

Generation of induced pluripotent stem cells (NIMHi027-A) from a Parkinson's disease patient harbouring a homozygous missense mutation for PLA2G6 gene (chr22:g.38112558C > T).

Stem cell research2026 Apr

A missense mutation in the PLA2G6 gene (chr22:g.38112558C > T) resulting in p.Arg741Gln substitution is implicated in young-onset Parkinson disease (YOPD), which presents with early motor symptoms clinically indistinguishable from other PD etiologies. Induced pluripotent stem cells (iPSCs) were generated from peripheral blood mononuclear cells of a PLA2G6 p.Arg741Gln patient with UPDRS-confirmed PD diagnosis using Sendai virus reprogramming technology. Complete characterization validated self-renewal capacity and pluripotency markers. This patient-derived iPSC line represents a valuable cellular model for mechanistic studies and drug discovery efforts targeting PLA2G6-associated Parkinsonism.

#2

Generation of induced pluripotent stem cells (NIMHi018-A) from a Parkinson's disease patient harbouring a heterozygous missense mutation for PINK1 variant c.1208G > A, p.Trp403Ter.

Stem cell research2025 Dec

The PTEN-induced kinase 1 (PINK1) gene mutation is the second most prevalent young-onset Parkinson disease (YOPD) characterized by early onset of motor symptoms that are often indistinguishable from other causes of PD. Induced pluripotent stem cells (iPSCs) were derived from peripheral blood mononuclear cells of a PD patient with PINK1 variant c.1208G > A, p.Trp403Ter using Sendai-virus reprogramming. PD diagnosis was confirmed via the Unified Parkinson's Disease Rating Scale (UPDRS). Characterization of the iPSC line ensured self-renewal and pluripotency. This resource serves as a valuable platform for drug screening and elucidating the pathophysiology of this mutation, facilitating advancements in PD research. PINK1 type of young-onset Parkinson disease is characterized by early onset (median age at onset 32 years) of tremor, bradykinesia, and rigidity that are often indistinguishable from other causes of Parkinson disease. Lower-limb dystonia may be a presenting sign. Postural instability, hyperreflexia, abnormal behavior, and psychiatric manifestations have been described. The disease is usually slowly progressive. Individuals have a marked and sustained response to oral administration of levodopa (L-dopa), frequently associated with L-dopa-induced fluctuations and dyskinesias. The diagnosis of PINK1 type of young-onset Parkinson disease is established by the identification of biallelic PINK1 pathogenic variants on molecular genetic testing. Treatment of manifestations: PINK1 type of young-onset Parkinson disease usually responds well to L-dopa and/or other dopamine agonists, which may be used in combination with catechol-O-methyltransferase inhibitors or monoamine oxidase-B inhibitors, anticholinergics, and amantadine. Physical therapy and/or occupational therapy to improve and/or maintain gross motor and fine motor skills as well as speech therapy. Invasive therapies include intrajejunal L-dopa-carbidopa pump, subcutaneous apomorphine pump, or deep brain stimulation. L-dopa-induced dyskinesias can be treated by reducing L-dopa dose, switching to dopamine receptor agonists, deep brain stimulation, or continuous treatment with L-dopa or apomorphine. Atypical neuroleptic agents can be used for neuropsychiatric manifestations; standard treatments for depression. Cholinesterase inhibitors can be used to treat dementia. Consider droxidopa, midodrine, fludrocortisone, and/or supportive measures for orthostasis. Symptomatic treatment for constipation. Surveillance: Neurologic evaluation every three to 12 months to assess motor and non-motor manifestations and treatment efficacy; in addition, assess for atypical manifestations and therapy needs at each visit. Neuropsychiatric evaluation in those with mood disorder / psychotic symptoms or as needed. Cognitive assessment annually or as needed. At each visit, assess nutrition and safety of feeding; assess for symptoms of orthostasis and measure supine and standing blood pressure and pulse; assess for constipation, urinary urgency, or urge incontinence; and assess family needs. Agents/circumstances to avoid: Neuroleptic treatment may exacerbate parkinsonism. PINK1 type of young-onset Parkinson disease is inherited in an autosomal recessive manner. If both parents are known to be heterozygous for a PINK1 pathogenic variant, each sib of an affected individual has at conception a 25% chance of being affected, a 50% chance of being heterozygous, and a 25% chance of inheriting neither of the familial PINK1 pathogenic variants. Once the PINK1 pathogenic variants have been identified in an affected family member, heterozygote testing for at-risk relatives and prenatal and preimplantation genetic testing are possible.

#3

Curcumin neuroprotective effects in Parkinson disease during pregnancy.

Brain research bulletin2023 Sep

Young onset Parkinson disease (YOPD) accounts for about 10% of PD patients, with the onset of symptoms between the ages of 21 and 40. At this age, the probability of pregnancy is high and there is a concern that the disease affects the fetuses. Therefore, in the present study, the effects of rotenone-induced PD on female mice as well as their fetuses and curcumin supplementation on the cerebral tissue of both female mice and their resulted fetuses were studied. The rotenone was injected subcutaneously to induce PD model of female mice. The different concentrations of curcumin were administrated every day i.p. for 3 weeks and the rotarod test was done on day 1 and 19. Cell viability was measured by MTT test and apoptosis and necrosis of cells were evaluate using flow cytometry technique. After primer design, the expressions of bax, bcl-2, miR-211 and circRNA 0001518 genes were measured using RT-PCR technique. Curcumin administration were improved cerebral cell viability of both female PD mice and resulted fetuses by preventing cell apoptosis and necrosis. bax, miR-211 and circRNA 0001518 were downregulated and bcl-2 overexpressed in cerebral neurons of PD mice and their fetuses. PD induction in mice affects their fetal brain, and curcumin can partially reduce the negative effects of PD on fetal brain cells by overexpressing bcl-2 and decreasing bax expression genes.

#4

Comparison of UPDRS III score between young and late onset Parkinson disease after deep brain stimulation: A meta-analysis.

Medicine2023 Nov 03

Parkinson disease (PD) is a common age-related neurodegenerative disease. Subthalamic nucleus deep brain stimulation (DBS) is a safe and effective surgical treatment for medically resistant advanced PD. However, the relationship between the age at PD onset and the efficacy of subthalamic nucleus DBS surgery remains unclear. Thus, we conducted a meta-analysis to compare motor symptom improvements after DBS for the treatment of young-onset and late-onset PD. We systematically searched the Medline and Embase databases (from inception to March 2023) for English publications. All published studies comparing the outcomes (Unified Parkinson's Disease Rating Scale part III [UPDRS III] scores) of DBS between the young-onset Parkinson disease (YOPD) and late-onset Parkinson disease (LOPD) groups were identified. The effect size was defined as the standardized mean difference (Hedge g) with 95% confidence intervals. The standardized mean difference was calculated by dividing the difference in UPDRS III scores between old and young patients by the pooled and weighted standard deviations. The meta-analysis was performed using R Statistical Software version 4.2.2 (The R Foundation for Statistical Computing). Six studies were eligible for inclusion. The standardized mean difference of UPDRS III score between young and old patients ranged from -0.54 to 1.43 with a pooled difference of 0.0932 (95% confidence intervals: - 0.4666 to 0.6530, I2 = 86.77%). Subgroup analyses were performed with a cutoff age of 65 years and did not show a significant difference in UPDRS III scores between patients with YOPD and LOPD (0.1877, -0.6663 to 1.0417). The efficacy of DBS in patients with YOPD and LOPD showed similar improvements in the UPDRS score; hence, DBS should be considered, if necessary, regardless of the onset age of PD.

#5

Parkin coregulates glutathione metabolism in adult mammalian brain.

Acta neuropathologica communications2023 Jan 23

We recently discovered that the expression of PRKN, a young-onset Parkinson disease-linked gene, confers redox homeostasis. To further examine the protective effects of parkin in an oxidative stress model, we first combined the loss of prkn with Sod2 haploinsufficiency in mice. Although adult prkn-/-//Sod2± animals did not develop dopamine cell loss in the S. nigra, they had more reactive oxidative species and a higher concentration of carbonylated proteins in the brain; bi-genic mice also showed a trend for more nitrotyrosinated proteins. Because these redox changes were seen in the cytosol rather than mitochondria, we next explored the thiol network in the context of PRKN expression. We detected a parkin deficiency-associated increase in the ratio of reduced glutathione (GSH) to oxidized glutathione (GSSG) in murine brain, PRKN-linked human cortex and several cell models. This shift resulted from enhanced recycling of GSSG back to GSH via upregulated glutathione reductase activity; it also correlated with altered activities of redox-sensitive enzymes in mitochondria isolated from mouse brain (e.g., aconitase-2; creatine kinase). Intriguingly, human parkin itself showed glutathione-recycling activity in vitro and in cells: For each GSSG dipeptide encountered, parkin regenerated one GSH molecule and was S-glutathionylated by the other (GSSG + P-SH [Formula: see text] GSH + P-S-SG), including at cysteines 59, 95 and 377. Moreover, parkin's S-glutathionylation was reversible by glutaredoxin activity. In summary, we found that PRKN gene expression contributes to the network of available thiols in the cell, including by parkin's participation in glutathione recycling, which involves a reversible, posttranslational modification at select cysteines. Further, parkin's impact on redox homeostasis in the cytosol can affect enzyme activities elsewhere, such as in mitochondria. We posit that antioxidant functions of parkin may explain many of its previously described, protective effects in vertebrates and invertebrates that are unrelated to E3 ligase activity.

Publicações recentes

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2026

Generation of induced pluripotent stem cells (NIMHi027-A) from a Parkinson's disease patient harbouring a homozygous missense mutation for PLA2G6 gene (chr22:g.38112558C > T).

Stem cell research
2025

Generation of induced pluripotent stem cells (NIMHi018-A) from a Parkinson's disease patient harbouring a heterozygous missense mutation for PINK1 variant c.1208G > A, p.Trp403Ter.

Stem cell research
2023

Comparison of UPDRS III score between young and late onset Parkinson disease after deep brain stimulation: A meta-analysis.

Medicine
2023

Curcumin neuroprotective effects in Parkinson disease during pregnancy.

Brain research bulletin
2023

Parkin coregulates glutathione metabolism in adult mammalian brain.

Acta neuropathologica communications
2023

The young-onset Parkinson disease.

Minerva medica
2022

Long-term efficacy of bilateral subthalamic deep brain stimulation in the parkinsonism of SCA 3: A rare case report.

European journal of neurology
2021

Age-associated insolubility of parkin in human midbrain is linked to redox balance and sequestration of reactive dopamine metabolites.

Acta neuropathologica
2020

Pathogenetic insights into young-onset Parkinson disease.

Nature reviews. Neurology
2018

Characteristic Motor and Nonmotor Symptoms Related to Quality of Life in Drug-Naïve Patients with Late-Onset Parkinson Disease.

Neuro-degenerative diseases
2017

Understanding uncertainty in young-onset Parkinson disease.

Chronic illness
2017

Four Cases of Parkinson Disease Diagnosed During the Postpartum Period.

Obstetrics and gynecology
2016

DOPA-sparing strategy in the treatment of young onset Parkinson's disease.

Journal of neurosciences in rural practice

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Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Generation of induced pluripotent stem cells (NIMHi027-A) from a Parkinson's disease patient harbouring a homozygous missense mutation for PLA2G6 gene (chr22:g.38112558C&#xa0;&gt;&#xa0;T).
    Stem cell research· 2026· PMID 41719744mais citado
  2. Generation of induced pluripotent stem cells (NIMHi018-A) from a Parkinson's disease patient harbouring a heterozygous missense mutation for PINK1 variant c.1208G &gt; A, p.Trp403Ter.
    Stem cell research· 2025· PMID 41075512mais citado
  3. Curcumin neuroprotective effects in Parkinson disease during pregnancy.
    Brain research bulletin· 2023· PMID 37543296mais citado
  4. Comparison of UPDRS III score between young and late onset Parkinson disease after deep brain stimulation: A meta-analysis.
    Medicine· 2023· PMID 37933060mais citado
  5. Parkin coregulates glutathione metabolism in adult mammalian brain.
    Acta neuropathologica communications· 2023· PMID 36691076mais citado
  6. Long-Term Outcomes of Subthalamic Nucleus and Globus Pallidus Interna Deep Brain Stimulation for Young-Onset Parkinson Disease.
    Neurol Clin Pract· 2026· PMID 41908734recente
  7. PINK1 Type of Young-Onset Parkinson Disease.
    · 1993· PMID 20301792recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:2828(Orphanet)
  2. MONDO:0017279(MONDO)
  3. GARD:16610(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Artigo Wikipedia(Wikipedia)
  7. Q11085(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.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Doença de Parkinson de início juvenil
Compêndio · Raras BR

Doença de Parkinson de início juvenil

ORPHA:2828 · MONDO:0017279
Prevalência
1-5 / 10 000
Herança
Autosomal recessive
CID-10
G20 · Doença de Parkinson
CID-11
Ensaios
4 ativos
Início
Adult
Prevalência
15.0 (Europe)
MedGen
UMLS
C0030567
EuropePMC
Wikidata
Wikipedia
Papers 10a
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