GENETIC DISEASES
Clinical trials for GENETIC DISEASES explained in plain language.
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Hope for duchenne: new drug targets rare gene mutation in phase 3 trial
Disease control Not yet recruitingThis study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…
Matched conditions: GENETIC DISEASES
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 22, 2026 12:03 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