FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 1
Clinical trials for FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 1 explained in plain language.
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Experimental gene 'Silencer' therapy enters human testing for rare muscle disease
Disease control Recruiting nowThis 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…
Matched conditions: FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Epicrispr Biotechnologies, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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New hope for rare muscle disease: experimental drug enters human trials
Disease control Recruiting nowThis study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will r…
Matched conditions: FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:02 UTC
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Could umbilical cord stem cells ease muscle weakness in FSHD?
Symptom relief Recruiting nowThis early-stage study tests whether stem cells from umbilical cord lining can safely help people with FSHD, a genetic muscle-weakening disease. Sixteen adults will receive two doses of the cells and two doses of a placebo (saline) through an IV, in random order. The main goal is…
Matched conditions: FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 1
Phase: PHASE1 • Sponsor: Restem, LLC. • Aim: Symptom relief
Last updated Jun 27, 2026 08:02 UTC