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autosomal recessive cerebellar ataxia (ARCA) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

autosomal recessive cerebellar ataxia

MONDO:0015244

Also known as:ARCAcerebellar ataxia, autosomal recessive

Find Clinical TrialsTrials

Overview

Autosomal recessive cerebellar ataxias (ARCA) are a heterogeneous group of rare neurological disorders involving both the central and peripheral nervous system (and in some cases other systems and organs), and characterized by degeneration or abnormal development of the cerebellum and spinal cord and, in most cases, early onset occurring before the age of 20 years.

OnsetMIDDLE_AGE, ADULT

Clinical Trials2 registered · 2 recruiting

Prognosis

Estimated prevalence: 1-9 in 100,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

2 clinical trials registered, 2 recruiting. Interventions under study include other interventions and gene therapy. Pipeline includes 1 NA. Research is primarily sponsored by academic and government institutions.

19 publications have been identified in PubMed for autosomal recessive cerebellar ataxia. Research spans Case Report / Case Series (37%), Basic Science / Preclinical (21%), and Review / Meta-Analysis (16%).

Research Type	Count	% of Total
Patient case studies	7

Data assembled from 4 of 12 sources · Last updated Sep 19, 2026, 11:57 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:1172

GARD

Genetic and Rare Diseases Info Center

GARD:18718

Questions for Your Care Team

→Are any of the 2 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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autosomal recessive cerebellar ataxia

Overview

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Recent Publications