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cerebellar ataxia - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

cerebellar ataxia

MONDO:0000437

Also known as:ataxia syndromecerebellar ataxiasspinocerebellar ataxia

Find Clinical TrialsTrials

Overview

A neurological syndrome characterized by clumsy and uncoordinated movement of the limbs, trunk, and cranial muscles. It results from pathology in the cerebellum and its connections, or in the proprioceptive sensory pathways.

TreatmentsNo approved treatments · 7 in pipeline

Clinical Trials121 registered · 61 recruiting

Diagnosis

Biomarker and diagnostic research for cerebellar ataxia has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for cerebellar ataxia. An additional 7 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for cerebellar ataxia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for cerebellar ataxia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

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.

121 clinical trials registered, 61 recruiting. Interventions under study include other interventions, drug therapy, medical devices, and gene therapy. Pipeline includes 9 PHASE3, 7 PHASE2, 11 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT02497534](https://clinicaltrials.gov/study/NCT02497534)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 3:00 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:102002

GARD

Genetic and Rare Diseases Info Center

GARD:19816

Questions for Your Care Team

→Are any of the 121 active clinical trials a good fit for my situation?
→With 7 compounds in the orphan drug pipeline, which are furthest along in development and which clinical trials should I be aware of?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Patient case studies	114	23%
Laboratory research	102	20%
Research summaries	99	20%
Disease patterns and progression	73	15%
Testing and diagnosis research	51	10%
Clinical study results	25	5%
New treatment approaches	19	4%
Other research	17	3%

Kisho

cerebellar ataxia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Uncovering Neuropathic Pain in CANVAS: Lessons from a Case-Based Literature Review.

Integrating remote testing and machine learning to identify markers of cerebellar ataxia at home.

Cerebellar Ataxia and Chorea: Genetic Etiologies and Key Considerations for Diagnosis and Management.

cerebellar ataxia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Uncovering Neuropathic Pain in CANVAS: Lessons from a Case-Based Literature Review.

Integrating remote testing and machine learning to identify markers of cerebellar ataxia at home.

Cerebellar Ataxia and Chorea: Genetic Etiologies and Key Considerations for Diagnosis and Management.