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
53 clinical trials for this condition and its sub-types.
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Experimental drug losmapimod tested in rare muscle disease – early hopes, but trial cut short
Disease control TerminatedThis study tested an experimental drug called losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was termina…
Phase: PHASE2 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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FSHD drug trial halted midway: what happened?
Disease control TerminatedThis study tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or…
Phase: PHASE3 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Experimental drug losmapimod tested for rare muscle disease
Disease control TerminatedThis phase 2 trial tested the drug losmapimod in 76 adults with FSHD, a genetic condition that causes progressive muscle weakness. Participants took either losmapimod or a placebo for 48 weeks to see if the drug was safe and could help control the disease. The study was terminate…
Phase: PHASE2 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Scientists gather leftover surgical tissue to unlock secrets of bone disease
Knowledge-focused TerminatedThis study collected bone, cartilage, skin, and fat tissue from 50 patients undergoing surgery at Children's National Medical Center. The goal was to grow cells from these tissues in the lab and analyze them to better understand bone and connective tissue diseases. The study was …
Sponsor: National Institute of Dental and Craniofacial Research (NIDCR) • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:52 UTC