MUSCULAR DYSTROPHIES
Clinical trials for MUSCULAR DYSTROPHIES explained in plain language.
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New hope for FSHD: Long-Term drug safety trial underway
Disease control OngoingThis study looks at the long-term safety and effectiveness of a drug called AOC 1020 for people with FSHD, a genetic disease that causes muscle weakness. About 84 adults who completed a previous study will receive the drug through an IV. The main goal is to check for side effects…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 17, 2026 13:54 UTC
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Experimental cell therapy aims to slow muscle loss in duchenne MD
Disease control OngoingThis phase 3 trial tests whether a cell therapy called deramiocel (CAP-1002) can slow muscle decline in boys and young men with Duchenne muscular dystrophy (DMD). About 106 participants receive either the therapy or a placebo every 3 months for a year, then all can receive the th…
Matched conditions: MUSCULAR DYSTROPHIES
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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Massive study aims to unlock secrets of rare childhood nerve diseases
Knowledge-focused OngoingThis study looks at people with inherited nerve and muscle disorders that start in childhood, like muscular dystrophy. Researchers want to learn how these diseases change over time and find their genetic causes. Participants include affected individuals, their family members, and…
Matched conditions: MUSCULAR DYSTROPHIES
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated May 17, 2026 13:59 UTC
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New model aims to speed up rare disease diagnoses
Knowledge-focused OngoingThis study is testing a new way to care for people with rare diseases. It brings together specialists, new technologies like genetic testing, and better coordination between hospitals and local services. The goal is to help 136 patients get a faster diagnosis and reduce unnecessa…
Matched conditions: MUSCULAR DYSTROPHIES
Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS • Aim: Knowledge-focused
Last updated May 17, 2026 13:57 UTC