Exon 44
Clinical trials for Exon 44 explained in plain language.
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New hope for duchenne: experimental drug targets genetic glitch
Disease control OngoingThis study tests an experimental drug called NS-089/NCNP-02 (Brogidirsen) in 20 boys with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping exon 44. The drug is given as a weekly IV infusion and aims to help the body produce a working version of the dyst…
Matched conditions: EXON 44
Phase 2 • Sponsor: NS Pharma, Inc. • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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Can a targeted infusion slow muscle decline in duchenne? a new trial aims to find out.
Disease control OngoingThis phase 2 trial tests an experimental drug called AOC 1044 in people with Duchenne muscular dystrophy whose genetic mutation makes them candidates for exon 44 skipping therapy. The drug is given by intravenous infusion. Researchers are evaluating long-term safety and whether i…
Matched conditions: EXON 44
Phase 2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Aug 01, 2026 00:00 UTC