GENETIC DISEASES
Clinical trials for GENETIC DISEASES explained in plain language.
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Promising new treatment for duchenne muscular dystrophy enters final testing phase
Disease control Not yet recruitingThis study tests an experimental drug called AOC 1044 (delpacibart zotadirsen) in 70 boys aged 7 to 16 with Duchenne muscular dystrophy who have a specific genetic change (exon 44 skipping). The drug is given through a vein and aims to improve muscle function, such as rising from…
Matched conditions: GENETIC DISEASES
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 27, 2026 11:48 UTC
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New study tracks rare genetic heart condition
Knowledge-focused Not yet recruitingThis study watches how dilated cardiomyopathy (a weakened heart muscle) progresses in adults with specific BAG3 gene changes. Researchers will track heart function and health events over time in 30 participants. No treatment is given—the goal is to better understand the disease.…
Matched conditions: GENETIC DISEASES
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated Jun 09, 2026 12:10 UTC