X-LINKED
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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: X-LINKED
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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Dental scans and AI could spot rare bone diseases faster
Knowledge-focused Not yet recruitingThis study will take 3D scans of the inside of the mouth from 240 people with rare bone or cartilage diseases and from healthy volunteers. Researchers will use shape analysis and artificial intelligence to see if these scans can help tell different diseases apart. The goal is to …
Matched conditions: X-LINKED
Phase: NA • Sponsor: University Hospital, Bordeaux • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:02 UTC