Early access to del-zota for DMD with exon 44 skipping
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This program provides early access to an experimental drug called del-zota (also known as AOC 1044) for people with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping exon 44. It is for those who have not had good results from other treatments and are willing to try a new approach.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a type of DMD that can be helped by skipping a part of the gene called exon 44.
- You need to be 2 years old or older.
- You must live in the United States and have a regular doctor here.
- If you have had gene therapy for DMD, it must be more than 12 months ago and it must not have worked well enough.
- You cannot have severe breathing or heart problems, or be near the end of life.
- You cannot be taking another experimental drug right now.
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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