Neuromuscular

Congenital muscular dystrophy due to dystroglycanopathy

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studies recruiting now

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studies registered in total

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countries with a recruiting site

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recruiting study posted to date

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Recruiting trials

Fetching live from ClinicalTrials.gov. This condition is not yet in our weekly snapshot; live results only.

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Patient organisations

We do not yet list a dedicated organisation for this condition. The directories below are the best route.

A group of conditions, not a single diagnosis

Orphanet classifies “Congenital muscular dystrophy due to dystroglycanopathy” as a category that groups several related conditions. Trials are usually registered under the specific condition, so the figures above may undercount. Browse the conditions in this group or see the classification on Orphanet.

Treatments being studied

5 approved treatments and 12 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Casimersen (Amondys 45)Approved: Viltolarsen (Viltepso)Approved: Golodirsen (Vyondys 53)Approved: Deflazacort (Calcort)Approved: Eteplirsen (Eteplirsen component of exondys 51)
Phase 3Drisapersen
Phase 3Metformin
Phase 2Trehalose
Phase 2Ataluren (Translarna)
Phase 2Domagrozumab
Phase 2Efmitermant Alfa
Phase 1/2Atyr-1940
Phase 1Omigapil

+ 4 more in development

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).