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Congenital

MONDO:0021140

A characteristic of a disease in which the disease is present at birth, regardless of cause.

Also known as: inborn

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

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Broader categories

Congenital or acquired (1) Disease characteristic (0)
Not yet recruiting 1 Completed 1
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  • 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

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