Facioscapulohumeral muscular dystrophy 1
MONDO:0008030Any facioscapulohumeral muscular dystrophy associated with contraction of the D4Z4 macrosatellite repeat in the subtelomeric region of chromosome 4q35.
Also known as: FSHD, FSHD1, FSHD1A, Landouzy-Dejerine muscular dystrophy, facioscapulohumeral muscular dystrophy 1, facioscapulohumeral muscular dystrophy 1A, facioscapulohumeral muscular dystrophy type 1, muscular dystrophy, facioscapulohumeral, type 1A
19 clinical trials for this condition and its sub-types.
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Experimental gene 'Silencer' therapy enters human testing for rare muscle disease
Disease control OngoingThis early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows a…
Phase: PHASE1, PHASE2 • Sponsor: Epicrispr Biotechnologies, Inc. • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC
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New hope for FSHD: Long-Term drug safety trial underway
Disease control OngoingThis study is for people with FSHD, a genetic disease that causes muscle weakness. It tests the long-term safety and how well the body tolerates a drug called AOC 1020, given through a vein. About 84 adults who completed a previous study will take part. The main goal is to check …
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jul 16, 2026 00:00 UTC