Raras
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Ataxia espinocerebelosa, tipo 6
ORPHA:98758CID-10 · G11.2CID-11 · 8A03.16OMIM 183086DOENÇA RARA

A Ataxia Espinocerebelar Tipo 6 (SCA6) é o subtipo mais comum da Ataxia Cerebelar Autossômica Dominante Tipo III (ADCA Tipo III), uma condição genética. Ela é caracterizada por surgir mais tarde na vida e progredir lentamente, causando dificuldade de coordenação ao andar e outros sinais que afetam o cerebelo, como problemas para coordenar os movimentos musculares e nistagmo (movimentos involuntários dos olhos).

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

A Ataxia Espinocerebelar Tipo 6 (SCA6) é o subtipo mais comum da Ataxia Cerebelar Autossômica Dominante Tipo III (ADCA Tipo III), uma condição genética. Ela é caracterizada por surgir mais tarde na vida e progredir lentamente, causando dificuldade de coordenação ao andar e outros sinais que afetam o cerebelo, como problemas para coordenar os movimentos musculares e nistagmo (movimentos involuntários dos olhos).

Pesquisas ativas
8 ensaios
13 total registrados no ClinicalTrials.gov
Publicações científicas
322 artigos
Último publicado: 2026 Mar 17

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-9 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Início
Adolescent
+ adult, elderly
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: G11.2
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (2)
0202010694
Sequenciamento completo do exoma (WES)genetic_test
0301070040
Atendimento em reabilitação — doenças rarasrehabilitation
Você se identifica com essa condição?
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

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Sinais e sintomas

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

Partes do corpo afetadas

🧠
Neurológico
9 sintomas
👁️
Olhos
3 sintomas
🫃
Digestivo
2 sintomas
💪
Músculos
1 sintomas
👂
Ouvidos
1 sintomas

+ 23 sintomas em outras categorias

Características mais comuns

100%prev.
Ataxia cerebelar progressiva
Muito frequente (99-80%)
100%prev.
Atrofia cerebelar
Obrigatório (100%)
90%prev.
Bradiopsia
Muito frequente (99-80%)
90%prev.
Marcha instável
Muito frequente (99-80%)
90%prev.
Instabilidade postural
Muito frequente (99-80%)
90%prev.
Ataxia da marcha
Muito frequente (99-80%)
39sintomas
Muito frequente (9)
Frequente (12)
Ocasional (12)
Sem dados (6)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 39 características clínicas mais associadas, ordenadas por frequência.

Ataxia cerebelar progressivaProgressive cerebellar ataxia
Muito frequente (99-80%)100%
Atrofia cerebelarCerebellar atrophy
Obrigatório (100%)100%
BradiopsiaBradyopsia
Muito frequente (99-80%)90%
Marcha instávelUnsteady gait
Muito frequente (99-80%)90%
Instabilidade posturalPostural instability
Muito frequente (99-80%)90%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico322PubMed
Últimos 10 anos125publicações
Pico201619 papers
Linha do tempo
2026Hoje · 2026🧪 2009Primeiro ensaio clínico📈 2016Ano 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

1 gene identificado com associação a esta condição. Padrão de herança: Autosomal dominant.

CACNA1AVoltage-dependent P/Q-type calcium channel subunit alpha-1ADisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Voltage-sensitive calcium channels (VSCC) mediate the entry of calcium ions into excitable cells and are also involved in a variety of calcium-dependent processes, including muscle contraction, hormone or neurotransmitter release, gene expression, cell motility, cell division and cell death. The isoform alpha-1A gives rise to P and/or Q-type calcium currents. P/Q-type calcium channels belong to the 'high-voltage activated' (HVA) group and are specifically blocked by the spider omega-agatoxin-IVA

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (2)
Presynaptic depolarization and calcium channel openingRegulation of insulin secretion
MECANISMO DE DOENÇA

Spinocerebellar ataxia 6

Spinocerebellar ataxia is a clinically and genetically heterogeneous group of cerebellar disorders. Patients show progressive incoordination of gait and often poor coordination of hands, speech and eye movements, due to degeneration of the cerebellum with variable involvement of the brainstem and spinal cord. SCA6 is an autosomal dominant cerebellar ataxia (ADCA), mainly caused by expansion of a CAG repeat in the coding region of CACNA1A. There seems to be a correlation between the repeat number and earlier onset of the disorder.

OUTRAS DOENÇAS (9)
migraine, familial hemiplegic, 1episodic ataxia type 2developmental and epileptic encephalopathy, 42spinocerebellar ataxia type 6
HGNC:1388UniProt:O00555

Variantes genéticas (ClinVar)

1,521 variantes patogênicas registradas no ClinVar.

🧬 CACNA1A: NM_001127222.2(CACNA1A):c.2056G>C (p.Gly686Arg) ()
🧬 CACNA1A: NM_001127222.2(CACNA1A):c.5084G>A (p.Cys1695Tyr) ()
🧬 CACNA1A: NM_001127222.2(CACNA1A):c.1782-1G>A ()
🧬 CACNA1A: NM_001127222.2(CACNA1A):c.4979G>T (p.Arg1660Leu) ()
🧬 CACNA1A: NM_001127222.2(CACNA1A):c.5081T>A (p.Val1694Asp) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 149 variantes classificadas pelo ClinVar.

53
96
Patogênica (35.6%)
VUS (64.4%)
VARIANTES MAIS SIGNIFICATIVAS
CACNA1A: NM_001127222.2(CACNA1A):c.2714_2721del (p.Ala905fs) [Likely pathogenic]
CACNA1A: NM_001127222.2(CACNA1A):c.1972dup (p.Met658fs) [Pathogenic]
CACNA1A: NM_001127222.2(CACNA1A):c.5414_5415del (p.Phe1805fs) [Likely pathogenic]
CACNA1A: NM_001127222.2(CACNA1A):c.5264A>G (p.Glu1755Gly) [Likely pathogenic]
CACNA1A: NM_001127222.2(CACNA1A):c.1237C>T (p.Gln413Ter) [Pathogenic]

Vias biológicas (Reactome)

2 vias biológicas associadas aos genes desta condição.

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 33
2Fase 22
·Pré-clínico8
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 13 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Ataxia espinocerebelosa, tipo 6

🗺️

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

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

Outros ensaios clínicos

13 ensaios clínicos encontrados, 8 ativos.

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

🥇Melhor nível de evidência: Revisão sistemática
Timeline de publicações
126 papers (10 anos)
#1

Quantitative susceptibility mapping of dentate nucleus iron in SCA6 and SCA31: comparison with pathological findings.

Journal of neurology2026 Mar 17

Spinocerebellar ataxia type 6 (SCA6) and 31 (SCA31) exhibit similar clinical and radiological features and have traditionally been distinguishable only through genetic testing. We focused on iron deposition in the cerebellar dentate nucleus (DN) to differentiate these diseases, referencing corresponding pathological findings. Using quantitative susceptibility mapping (QSM), DN susceptibility was measured in 32 patients with SCA6, 31 with SCA31, and 37 controls, and the values were compared among groups. Correlations between susceptibility and disease duration or Scale for the Assessment and Rating of Ataxia (SARA) scores were also evaluated. In separate autopsy cases, Berlin blue and anti-ferritin immunostaining were performed on the DN in five SCA6 cases, one SCA31 case, and three controls. Susceptibility was significantly lower in patients with SCA6 than in those with SCA31 or controls. In SCA6, susceptibility inversely correlated with disease duration, whereas no such correlations were observed in SCA31. In contrast, no significant correlation was noted between susceptibility and SARA scores in either SCA6 or SCA31. Pathological findings showed absent ferritin staining in SCA6, strong staining in controls, and intermediate staining in SCA31. Berlin blue staining was negative in all groups. Reduced DN susceptibility in SCA6 reflects ferritin loss, distinguishing it from SCA31. Assessing DN susceptibility using QSM or SWI may provide useful imaging markers to complement the diagnosis of SCA6 and SCA31.

#2

Resilience to Endoplasmic Reticulum Stress Mitigates Membrane Hyperexcitability Underlying Late Disease Onset in a Murine Model of SCA6.

Annals of neurology2026 Feb

An enduring puzzle in many inherited neurological disorders is the late onset of symptoms despite expression of function-impairing mutant protein early in life. We examined the basis for onset of impairment in spinocerebellar ataxia type 6 (SCA6), a canonical late-onset neurodegenerative ataxia which results from a polyglutamine expansion in the voltage gated calcium channel, Cav2.1. We performed serial transcriptome analysis with weighted gene correlation network analysis to investigate mechanisms for resilience in SCA6 mice that prevent onset of symptoms. We examined changes in membrane excitability that result in cerebellar Purkinje neuron spiking abnormalities through patch-clamp recordings of Purkinje neurons in acute brain slices. Using unbiased transcriptome analysis, we identified endoplasmic reticulum (ER) stress as a driver of disease. Using spatial transcriptome analysis, we identified Purkinje neuron specific changes in unfolded protein response (UPR) related pathways. Novel activation of a store-operated calcium current due to ER stress is the cause for Purkinje neuron spiking abnormalities in SCA6 mice. The impairments in Purkinje neuron spiking are unrelated to Cav2.1 ion-flux function. Redundant pathways of the UPR act through a HSP90-dependent mechanism to mitigate this ER stress. Our studies support a model whereby proteotoxicity from misfolded mutant Cav2.1 is mitigated by a HSP90-dependent UPR, and age-related breakdown of this response causes motor dysfunction and aberrant Purkinje neuron spiking. These studies elucidate a mechanism of resilience connecting aberrant proteostasis and calcium-dependent intrinsic membrane hyperexcitability to explain delayed disease onset more widely in age-dependent neurodegenerative disease. ANN NEUROL 2026;99:502-522.

#3

Spinocerebellar Ataxia Type 6 Initially Diagnosed as Alcoholic Cerebellar Degeneration: A Case Report.

Internal medicine (Tokyo, Japan)2026 Feb 10

Spinocerebellar ataxia type 6 (SCA6) is an autosomal dominant adult-onset neurodegenerative disorder. Cases may appear sporadic when the family history is uninformative or relatives' symptoms are unrecognized as disease-related. We herein report a 49-year-old male with no family history of ataxia but with excessive alcohol consumption, initially diagnosed with alcoholic cerebellar degeneration (ACD) and subsequently identified as having SCA6. Diagnosing ACD is difficult because reliable diagnostic markers and threshold levels of alcohol intake predicting cerebellar degeneration are lacking. Alternative diagnoses, including SCA6, should be considered when clinical or imaging findings are atypical and the family history is uninformative.

#4

A diagnostic challenge: spinocerebellar ataxia type 6 presenting with dystonia and parkinsonism.

BMJ case reports2025 Aug 22

Spinocerebellar ataxias (SCA) are a group of hereditary cerebellar ataxias that are autosomal dominant. They often manifest as an adult-onset progressive neurodegenerative disease with predominantly cerebellar features of gait ataxia, nystagmus and dysarthria. SCA6 is a subtype of SCA which has been historically classified as 'pure cerebellar'. However, many patients may still present with non-cerebellar features. We present a woman in her 70s who was referred to a movement disorder clinic with rigidity, dystonia, upper limb contractures, dysarthria, ocular disturbance and muscle atrophy, with a strong family history of affected individuals. Whole exome sequencing identified 22 CAG repeats in the CACNA1A gene, in keeping with SCA6. A review of the literature identified a broader SCA6 phenotype, which can present with non-cerebellar features, and present a diagnostic challenge.

#5

Redefining the Pathogenic CAG Repeat Units Threshold in CACNA1A for Spinocerebellar Ataxia Type 6.

Neurology. Genetics2025 Apr

Spinocerebellar ataxia type 6 (SCA6) is caused by expansion of CAG repeat units (RUs) in CACNA1A. While the pathologic threshold has been considered to be 20 or 21 RUs, the lower limit remains controversial. This study aimed to clarify the pathologic significance of RUs in SCA6, including the role of opposite alleles (OAs). This was an observational study of patients with suspected spinocerebellar ataxia who underwent SCA6 genetic testing. We analyzed the relationship between CACNA1A RUs and age at onset (AAO). Family history positivity rates were examined for different RUs of the expanded allele (EA). Regression analyses were performed for AAO estimation based on the EA RUs. The influence of OAs on AAO was investigated, particularly in cases with 21-22 EA RUs. In total, 2,768 participants were enrolled. Family history positivity rates increased progressively above 19 RUs and plateaued at ≥23 RUs. Regression analysis of cases with ≥23 RUs showed that 96.20% of cases with ≥23 RUs, 90.67% of cases with 22 RUs, 91.15% of cases with 21 RUs, 61.54% of cases with 20 RUs, and 33.33% of cases with 19 RUs fell within the 95% prediction interval for AAO. However, no patients with ≤18 RUs were included. In the 21-22 RU group, OAs significantly influenced AAO, and ≥17 RUs had a significant effect. For ≥23 RUs, no significant OA effect was observed. Cases with 19-20 RUs showed a higher prevalence of OA with ≥19 RUs compared with cases with ≥23 RUs. Our findings suggest that clinical manifestation within a typical lifespan likely requires at least 19 RUs. The 19-20 RU range represents an intermediate zone where OA may influence disease likelihood. For 21-22 RUs, OA significantly affects AAO, indicating a complex interplay between EA and OA. ≥23 RUs seem sufficient to cause disease onset within a typical lifespan, regardless of OA. These results provide a new paradigm for SCA6 diagnosis and genetic counseling, emphasizing the need for cautious interpretation of the intermediate RU range and consideration of OA.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC186 artigos no totalmostrando 122

2026

Quantitative susceptibility mapping of dentate nucleus iron in SCA6 and SCA31: comparison with pathological findings.

Journal of neurology
2026

Spinocerebellar Ataxia Type 6 Initially Diagnosed as Alcoholic Cerebellar Degeneration: A Case Report.

Internal medicine (Tokyo, Japan)
2025

Commentary on the Flocculonodular Lobe as a Cerebellar Resilience Zone: Could Exercise Really be the Best Medicine?

Cerebellum (London, England)
2026

Resilience to Endoplasmic Reticulum Stress Mitigates Membrane Hyperexcitability Underlying Late Disease Onset in a Murine Model of SCA6.

Annals of neurology
2025

Stimulation of Purkinje cell firing reverses early onset spatial navigation deficits in a spinocerebellar ataxia type 6 mouse model.

Neurobiology of disease
2025

A diagnostic challenge: spinocerebellar ataxia type 6 presenting with dystonia and parkinsonism.

BMJ case reports
2025

Homozygous CAG Repeat Expansion in Spinocerebellar Ataxia Type 6: Longitudinal Analysis of Vestibulo-Ocular Reflex Findings.

Cerebellum (London, England)
2025

Effects of Levetiracetam on Episodic Ataxia Type 2 and Spinocerebellar Ataxia Type 6 with Episodic Ataxic Symptoms: A Case Series.

Genes
2025

Redefining the Pathogenic CAG Repeat Units Threshold in CACNA1A for Spinocerebellar Ataxia Type 6.

Neurology. Genetics
2025

Resilience to Endoplasmic Reticulum Stress Mitigates Calcium-Dependent Membrane Hyperexcitability Underlying Late Disease Onset in SCA6.

bioRxiv : the preprint server for biology
2024

L-arginine in patients with spinocerebellar ataxia type 6: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.

EClinicalMedicine
2024

Quantitative Oculomotor and Vestibular Profile in Spinocerebellar Ataxia Type 6 - Systematic Review and Meta-Analysis.

Cerebellum (London, England)
2024

Cerebellar type 1 metabotropic glutamate receptor availability decreases with disease progression in spinocerebellar ataxia type 6.

Journal of the neurological sciences
2024

Intrafamilial neurological phenotypic variability due to either biallelic or monoallelic pathogenic variants in CACNA1A.

Frontiers in neurology
2024

CACNA1A variant associated with generalized dystonia.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2024

Developing a pathway to clinical trials for CACNA1A-related epilepsies: A patient organization perspective.

Therapeutic advances in rare disease
2024

Phenotypic analysis of ataxia in spinocerebellar ataxia type 6 mice using DeepLabCut.

Scientific reports
2024

Mitochondrial damage and impaired mitophagy contribute to disease progression in SCA6.

Acta neuropathologica
2024

Subcellular localization and ER-mediated cytotoxic function of α1A and α1ACT in spinocerebellar ataxia type 6.

Biochemical and biophysical research communications
2024

Mild Deficits in Fear Learning: Evidence from Humans and Mice with Cerebellar Cortical Degeneration.

eNeuro
2023

Endosomal dysfunction contributes to cerebellar deficits in spinocerebellar ataxia type 6.

eLife
2023

Spinocerebellar Ataxia Type 6 and Japanese Immigration to Brazil.

Movement disorders clinical practice
2023

Dopa-responsive dystonia in spinocerebellar ataxia 6: A case report.

Clinical neurology and neurosurgery
2023

Age-related differences of cerebellar cortex and nuclei: MRI findings in healthy controls and its application to spinocerebellar ataxia (SCA6) patients.

NeuroImage
2022

The role of the basal ganglia and cerebellum in adaptation to others' speech rate and rhythm: A study of patients with Parkinson's disease and cerebellar degeneration.

Cortex; a journal devoted to the study of the nervous system and behavior
2022

Activation of TrkB-Akt signaling rescues deficits in a mouse model of SCA6.

Science advances
2022

Loss of Flocculus Purkinje Cell Firing Precision Leads to Impaired Gaze Stabilization in a Mouse Model of Spinocerebellar Ataxia Type 6 (SCA6).

Cells
2022

Cerebellar glutamatergic system impacts spontaneous motor recovery by regulating Gria1 expression.

NPJ Regenerative medicine
2022

Quantitative susceptibility mapping of basal ganglia iron is associated with cognitive and motor functions that distinguish spinocerebellar ataxia type 6 and type 3.

Frontiers in neuroscience
2022

Cerebellar contribution to threat probability in a SCA6 mouse model.

Human molecular genetics
2022

Criteria-unfulfilled multiple system atrophy at an initial stage exhibits laterality of middle cerebellar peduncles.

Journal of the neurological sciences
2022

The complexities of CACNA1A in clinical neurogenetics.

Journal of neurology
2022

Efficacy of nilotinib in monozygotic twins with spinocerebellar ataxia type 6.

Journal of neurology
2021

Mutations in the Voltage Dependent Calcium Channel CACNA1A (P/Q type alpha 1A subunit) Causing Neurological Disorders - An Overview.

Neurology India
2021

Novel Mutation in CACNA1A Associated with Activity-Induced Dystonia, Cervical Dystonia, and Mild Ataxia.

Case reports in neurological medicine
2023

The Electrophysiological Findings in Spinocerebellar Ataxia Type 6: Evidence From 24 Patients.

Journal of clinical neurophysiology : official publication of the American Electroencephalographic Society
2021

Spinocerebellar ataxia type 6 presenting with hallucination.

Psychogeriatrics : the official journal of the Japanese Psychogeriatric Society
2021

The electrophysiological footprint of CACNA1A disorders.

Journal of neurology
2020

Spinocerebellar Ataxia Type 6: A Disorder of Connectivity?

The Journal of neuroscience : the official journal of the Society for Neuroscience
2021

Commentary to: "The Pathophysiology and Clinical Manifestations of Spinocerebellar Ataxia Type 6" by Rentiya et al., Cerebellum 2020;19(3):459-464).

Cerebellum (London, England)
2020

Blended phenotype of adult-onset Alexander disease and spinocerebellar ataxia type 6.

Neurology. Genetics
2021

New Nonsense Variant c.2983G>T; p.Glu995* in the CACNA1A Gene Causes Progressive Autosomal Dominant Ataxia.

Journal of movement disorders
2020

Spinocerebellar ataxia type 6 family with phenotypic overlap with Multiple System Atrophy.

Neurologia i neurochirurgia polska
2020

Feasibility and Acceptability of Lee Silverman Voice Treatment in Progressive Ataxias.

Cerebellum (London, England)
2020

Association of A Novel Splice Site Mutation in P/Q-Type Calcium Channels with Childhood Epilepsy and Late-Onset Slowly Progressive Non-Episodic Cerebellar Ataxia.

International journal of molecular sciences
2020

Dopaminergic function in spinocerebellar ataxia type 6 patients with and without parkinsonism.

Journal of neurology
2020

Two distinct phenotypes, hemiplegic migraine and episodic Ataxia type 2, caused by a novel common CACNA1A variant.

BMC neurology
2020

Cerebral Venous Thrombosis: An Unexpected Complication with Cerebrospinal Fluid Leaks after a Fall in a Patient with Spinocerebellar Ataxia Type 6.

Internal medicine (Tokyo, Japan)
2020

Generation of induced pluripotent stem cell line (ZZUi0018-A ) from a patient with spinocerebellar ataxia type 6.

Stem cell research
2020

The Pathophysiology and Clinical Manifestations of Spinocerebellar Ataxia Type 6.

Cerebellum (London, England)
2020

Evolution of the vestibular function during head impulses in spinocerebellar ataxia type 6.

Journal of neurology
2020

Temporal Invariance in SCA6 Is Related to Smaller Cerebellar Lobule VI and Greater Disease Severity.

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

Impaired Adaptive Motor Learning Is Correlated With Cerebellar Hemispheric Gray Matter Atrophy in Spinocerebellar Ataxia Patients: A Voxel-Based Morphometry Study.

Frontiers in neurology
2019

Action perception recruits the cerebellum and is impaired in patients with spinocerebellar ataxia.

Brain : a journal of neurology
2019

Quantifying iron deposition in the cerebellar subtype of multiple system atrophy and spinocerebellar ataxia type 6 by quantitative susceptibility mapping.

Journal of the neurological sciences
2019

Prediction of Survival With Long-Term Disease Progression in Most Common Spinocerebellar Ataxia.

Movement disorders : official journal of the Movement Disorder Society
2019

Degenerative and acquired sporadic adult onset ataxia.

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

α1ACT Is Essential for Survival and Early Cerebellar Programming in a Critical Neonatal Window.

Neuron
2019

Beneficial effects of cerebellar rTMS stimulation on a patient with spinocerebellar ataxia type 6.

Brain stimulation
2018

Transcranial magnetic stimulation for diplopia in a patient with spinocerebellar ataxia type 6: a case report.

Cerebellum &amp; ataxias
2018

Cerebellar Degeneration Increases Visual Influence on Dynamic Estimates of Verticality.

Current biology : CB
2019

Analysis of CACNA1A CAG repeat lengths in patients with familial ALS.

Neurobiology of aging
2019

Major intra-familial phenotypic heterogeneity and incomplete penetrance due to a CACNA1A pathogenic variant.

European journal of medical genetics
2019

The neuropsychiatric phenotype in CACNA1A mutations: a retrospective single center study and review of the literature.

European journal of neurology
2018

Author Correction: Sensorimotor adaptation as a behavioural biomarker of early spinocerebellar ataxia type 6.

Scientific reports
2018

The benefits of a Neurogenetics clinic in an adult Academic Teaching Hospital.

Irish journal of medical science
2018

Molecular genetic testing for hereditary ataxia: What every neurologist should know.

Neurology. Clinical practice
2018

A refractory head tremor appearing after volatile anesthesia combined with epidural anesthesia in a patient with spinocerebellar ataxia type 6.

JA clinical reports
2018

Targeting the CACNA1A IRES as a Treatment for Spinocerebellar Ataxia Type 6.

Cerebellum (London, England)
2018

Sequencing analysis of the SCA6 CAG expansion excludes an influence of repeat interruptions on disease onset.

Journal of neurology, neurosurgery, and psychiatry
2017

Distinct cerebellar regions related to motor and cognitive performance in SCA6 patients.

Neuropsychologia
2017

Trinucleotide repeat disorders.

Handbook of clinical neurology
2018

Selective Patterns of Cognitive Impairment in Spinocerebellar Ataxia Type 6 and Idiopathic Late-Onset Cerebellar Ataxia.

Archives of clinical neuropsychology : the official journal of the National Academy of Neuropsychologists
2017

Bicistronic CACNA1A Gene Expression in Neurons Derived from Spinocerebellar Ataxia Type 6 Patient-Induced Pluripotent Stem Cells.

Stem cells and development
2017

Genetic Screening for Spinocerebellar Ataxia Genes in a Japanese Single-Hospital Cohort.

Journal of movement disorders
2017

Sensorimotor adaptation as a behavioural biomarker of early spinocerebellar ataxia type 6.

Scientific reports
2017

Alternative splicing in the C-terminal tail of Cav2.1 is essential for preventing a neurological disease in mice.

Human molecular genetics
2017

Downbeat nystagmus as an initial clinical sign in spinocerebellar ataxia type 6.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2017

Spinocerebellar ataxia type 6 presenting with parkinsonism, pre-synaptic dopaminergic dysfunction and hyperechogenicity of the substantia nigra.

Journal of the neurological sciences
2017

Resting-state functional connectivity and cognitive dysfunction correlations in spinocerebelellar ataxia type 6 (SCA6).

Human brain mapping
2017

Gene dosage effect in spinocerebellar ataxia type 6 homozygotes: A clinical and neuropathological study.

Journal of the neurological sciences
2017

Vulnerability of Purkinje Cells Generated from Spinocerebellar Ataxia Type 6 Patient-Derived iPSCs.

Cell reports
2017

Progression of Dysphagia in Spinocerebellar Ataxia Type 6.

Dysphagia
2016

Polyglutamine length-dependent toxicity from α1ACT in Drosophila models of spinocerebellar ataxia type 6.

Biology open
2017

Cognitive dysfunction in patients with spinocerebellar ataxia type 6.

Journal of neurology
2016

Transient Developmental Purkinje Cell Axonal Torpedoes in Healthy and Ataxic Mouse Cerebellum.

Frontiers in cellular neuroscience
2016

Validity of a wearable accelerometer to quantify gait in spinocerebellar ataxia type 6.

Physiological measurement
2016

Impaired Spatio-Temporal Predictive Motor Timing Associated with Spinocerebellar Ataxia Type 6.

PloS one
2016

Spinocerebellar ataxia type 6 in eastern India: Some new observations.

Annals of Indian Academy of Neurology
2016

Evaluation of parkinsonism and striatal dopamine transporter loss in patients with spinocerebellar ataxia type 6.

Journal of neurology
2017

Transient cerebellar alterations during development prior to obvious motor phenotype in a mouse model of spinocerebellar ataxia type 6.

The Journal of physiology
2016

Relationship between type 1 metabotropic glutamate receptors and cerebellar ataxia.

Journal of neurology
2016

[Missense mutation R1345Q in CACNA1A gene causes a new type of ataxia with episodic tremor: clinical features, genetic analysis and treatment in a familial case].

Nan fang yi ke da xue xue bao = Journal of Southern Medical University
2016

An miRNA-mediated therapy for SCA6 blocks IRES-driven translation of the CACNA1A second cistron.

Science translational medicine
2016

4-aminopyridine reverses ataxia and cerebellar firing deficiency in a mouse model of spinocerebellar ataxia type 6.

Scientific reports
2016

Polysomnography findings in spinocerebellar ataxia type 6.

Journal of sleep research
2017

Sensory and motor cortex function contributes to symptom severity in spinocerebellar ataxia type 6.

Brain structure &amp; function
2016

Biallelic CACNA1A mutations cause early onset epileptic encephalopathy with progressive cerebral, cerebellar, and optic nerve atrophy.

American journal of medical genetics. Part A
2016

Next-generation sequencing identifies novel CACNA1A gene mutations in episodic ataxia type 2.

Molecular genetics &amp; genomic medicine
2016

Large-Scale Functional RNAi Screen in C. elegans Identifies TGF-β and Notch Signaling Pathways as Modifiers of CACNA1A.

ASN neuro
2016

Eye movement disorders are an early manifestation of CACNA1A mutations in children.

Developmental medicine and child neurology
2015

Rapid Onset of Motor Deficits in a Mouse Model of Spinocerebellar Ataxia Type 6 Precedes Late Cerebellar Degeneration.

eNeuro
2014

An altered GABA-A receptor function in spinocerebellar ataxia type 6 and familial hemiplegic migraine type 1 associated with the CACNA1A gene mutation.

BBA clinical
2016

Structural and Functional Magnetic Resonance Imaging of the Cerebellum: Considerations for Assessing Cerebellar Ataxias.

Cerebellum (London, England)
2016

SCA 6 with Writer's Cramp: The Phenotype Expanded.

Movement disorders clinical practice
2016

Early Cerebellar Network Shifting in Spinocerebellar Ataxia Type 6.

Cerebral cortex (New York, N.Y. : 1991)
2015

Spinocerebellar ataxia type 6 protein aggregates cause deficits in motor learning and cerebellar plasticity.

The Journal of neuroscience : the official journal of the Society for Neuroscience
2015

Decreased metabotropic glutamate receptor type 1 availability in a patient with spinocerebellar ataxia type 6: A (11)C-ITMM PET study.

Journal of the neurological sciences
2015

Loss of MyD88 alters neuroinflammatory response and attenuates early Purkinje cell loss in a spinocerebellar ataxia type 6 mouse model.

Human molecular genetics
2015

Clinical characteristics of combined cases of spinocerebellar ataxia types 6 and 31.

Journal of neurogenetics
2015

Familial hemiplegic migraine type 1 associated with parkinsonism: a case report.

Case reports in neurology
2015

DnaJ-1 and karyopherin α3 suppress degeneration in a new Drosophila model of Spinocerebellar Ataxia Type 6.

Human molecular genetics
2015

Force dysmetria in spinocerebellar ataxia 6 correlates with functional capacity.

Frontiers in human neuroscience
2015

[SCA6 presenting parkinsonism without ataxia--A case report].

Rinsho shinkeigaku = Clinical neurology
2015

Sensorimotor processing for balance in spinocerebellar ataxia type 6.

Movement disorders : official journal of the Movement Disorder Society
2015

Structural and functional MRI abnormalities of cerebellar cortex and nuclei in SCA3, SCA6 and Friedreich's ataxia.

Brain : a journal of neurology
2015

Molecular mechanism of Spinocerebellar Ataxia type 6: glutamine repeat disorder, channelopathy and transcriptional dysregulation. The multifaceted aspects of a single mutation.

Frontiers in cellular neuroscience
2015

Segmentation of the Cerebellar Peduncles Using a Random Forest Classifier and a Multi-object Geometric Deformable Model: Application to Spinocerebellar Ataxia Type 6.

Neuroinformatics
2015

The 3-second rule in hereditary pure cerebellar ataxia: a synchronized tapping study.

PloS one
2015

Rare frequency of downbeat positioning nystagmus in spinocerebellar ataxia type 31.

Journal of the neurological sciences
2015

Vestibular Performance During High-Acceleration Stimuli Correlates with Clinical Decline in SCA6.

Cerebellum (London, England)
2015

Molecular characterization and functional expression of the Apis mellifera voltage-dependent Ca2+ channels.

Insect biochemistry and molecular biology
Ver todos os 186 no EuropePMC

Associações

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Comunidades

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

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. Quantitative susceptibility mapping of dentate nucleus iron in SCA6 and SCA31: comparison with pathological findings.
    Journal of neurology· 2026· PMID 41843260mais citado
  2. Resilience to Endoplasmic Reticulum Stress Mitigates Membrane Hyperexcitability Underlying Late Disease Onset in a Murine Model of SCA6.
    Annals of neurology· 2026· PMID 40990218mais citado
  3. Spinocerebellar Ataxia Type 6 Initially Diagnosed as Alcoholic Cerebellar Degeneration: A Case Report.
    Internal medicine (Tokyo, Japan)· 2026· PMID 41672535mais citado
  4. A diagnostic challenge: spinocerebellar ataxia type 6 presenting with dystonia and parkinsonism.
    BMJ case reports· 2025· PMID 40846501mais citado
  5. Redefining the Pathogenic CAG Repeat Units Threshold in CACNA1A for Spinocerebellar Ataxia Type 6.
    Neurology. Genetics· 2025· PMID 39996131mais citado
  6. Commentary on the Flocculonodular Lobe as a Cerebellar Resilience Zone: Could Exercise Really be the Best Medicine?
    Cerebellum· 2025· PMID 41247627recente
  7. Stimulation of Purkinje cell firing reverses early onset spatial navigation deficits in a spinocerebellar ataxia type 6 mouse model.
    Neurobiol Dis· 2025· PMID 40976063recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:98758(Orphanet)
  2. OMIM OMIM:183086(OMIM)
  3. MONDO:0008457(MONDO)
  4. GARD:10351(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q2868788(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

Ataxia espinocerebelosa, tipo 6
Compêndio · Raras BR

Ataxia espinocerebelosa, tipo 6

ORPHA:98758 · MONDO:0008457
Prevalência
1-9 / 1 000 000
Herança
Autosomal dominant
CID-10
G11.2 · Ataxia cerebelar de início tardio
CID-11
Ensaios
8 ativos
Início
Adolescent, Adult, Elderly
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0752124
Repurposing
1 candidato
taltirelinthyrotropin releasing hormone receptor agonist
EuropePMC
Wikidata
Papers 10a
Evidência
🥇 Rev. sistemática
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