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Drugs in development · updated 30 October 2026

Limb-girdle muscular dystrophy: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:263

As of 30 October 2026 there are 4 active clinical trials (recruiting or ongoing, phases I–III) on 3 drugs for limb-girdle muscular dystrophy. 3 are experimental, that is not yet approved in the United States.

4Active trials
3Experimental drugs
1Experimental, 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
SRP-9003III2NCT06246513
AB-1003I–II1NCT05230459
BBP-418II1NCT04800874

Sorted by most advanced phase and number of active trials.

All 3 drugs, with the details

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Broader categories

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: D049288 “Muscular Dystrophies, Limb-Girdle”. Prevalence: Orphanet.

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