RecruitingNCT05989620
Neuromuscular
Congenital muscular dystrophy type 1D
2
studies recruiting now
as of 7 Sept 2026
4
studies registered in total
as of 7 Sept 2026
1
countries with a recruiting site
as of 7 Sept 2026
14 Aug 2023
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT05902351
Natural History Study for Charcot Marie Tooth Disease
Showing the 2 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
Search all Congenital muscular dystrophy type 1D studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Patient organisations
We do not yet list a dedicated organisation for this condition. The directories below are the best route.
About Congenital muscular dystrophy type 1D
RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations.
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).