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Congenital or acquired

MONDO:0021139

2 clinical trials for this condition and its sub-types.

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Sub-types

Congenital (1) Acquired (0)

Broader categories

Disease characteristic (0)

Not yet recruiting 1 Completed 1

Hope for duchenne: new drug targets rare gene mutation in phase 3 trial

Disease control Not yet recruiting

This 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…

Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control

Last updated Jun 27, 2026 12:32 UTC