Exon 44 skipping drug for Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new drug (NS-089/NCNP-02) that aims to help boys with Duchenne muscular dystrophy produce a shorter but still working version of the dystrophin protein. It's for boys who are still walking and have a specific genetic type that can be fixed by skipping a part of the gene called exon 44.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Boys age 4 to 14 years old
- Must have a confirmed DMD gene mutation that can be fixed by skipping exon 44 (a specific genetic target)
- Able to walk independently (no walker, crutches, or wheelchair)
- Can stand up from the floor without help in under 20 seconds
- On a stable dose of steroids for at least 3 months
- Weigh at least 20 kg (44 lbs) if screening for Part 1
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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