X-LINKED
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Hope for duchenne: new drug targets rare gene mutation in phase 3 trial
Disease control Recruiting nowThis 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: X-LINKED
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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New study tracks hemophilia a risks in mothers and babies
Knowledge-focused Recruiting nowThis study follows 500 mother-child pairs where the mother has a severe hemophilia A gene. It aims to understand bleeding risks during pregnancy and childbirth for mothers, and how children develop antibodies to factor VIII treatment. By observing families over time, researchers …
Matched conditions: X-LINKED
Sponsor: University of Washington • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC