Testing a New Therapy for DMD with Exon 44 Mutations
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a boy between 7 and 16 years old
- You have been diagnosed with DMD and have genetic testing confirming an exon 44 mutation
- You are still able to walk (ambulatory)
- You have been taking steroid medication at a steady dose for at least 6 months
- You have not received gene therapy or cell therapy before
- You have not received another oligonucleotide treatment (a type of genetic medicine) in the past 6 months
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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