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Drugs in development · updated 30 October 2026

Spinocerebellar ataxia: drugs in development

As of 30 October 2026 there are 5 active clinical trials (recruiting or ongoing, phases I–III) on 4 drugs for spinocerebellar ataxia. 4 are experimental, that is not yet approved in the United States.

5Active trials
4Experimental drugs
2Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
TroriluzoleBHV-4157, Dazluma®III2NCT03701399
N-Acetyl-L-LeucineIII1NCT07221292
StemchymalII1NCT06397274
VO659I–II1NCT05822908

Sorted by most advanced phase and number of active trials.

All 4 drugs, with the details

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Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D020754 “Spinocerebellar Ataxias”.

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