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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, 1 tagged with Congenital itself.

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Part of

↑ Congenital or acquired (2)

Including sub-types (2) Tagged with Congenital (1)

Trials to join now! 1 Completed 1

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

Disease control Recruiting now

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 3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control

Last updated Sep 17, 2026 00:00 UTC