Testing WVE-N531 for Duchenne muscular dystrophy exon 53
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This early-phase study tests WVE-N531 in people with Duchenne muscular dystrophy (DMD) whose genetics specifically match an “exon 53” change. It may help by targeting the muscle disease process, and it includes procedures like muscle biopsies and regular heart/lung checks.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) have a confirmed diagnosis of Duchenne muscular dystrophy (DMD).
- Your DMD genetic result shows a mutation that can be treated with an exon 53 approach.
- You are male, and you can be walking or not walking depending on which part of the study you enter.
- You can do study muscle biopsies, ideally from the deltoid (shoulder) muscle.
- Heart and lung tests are stable enough for participation (specific breathing and heart pumping thresholds).
- If you take steroid medicine for DMD, the dose must have stayed the same for several months before screening.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
This trial tests a new gene therapy called SGT-003 in boys and men with Duchenne muscular dystrophy who are still able to walk. The treatment aims to deliver a working version of the dystrophin gene to help muscle cells produce the protein they are missing.
This study tests a gene therapy that aims to help muscles produce a protein they are missing. It is for people with Duchenne muscular dystrophy who are no longer able to walk, and it may help slow the progression of the disease.
This trial is for people who have already taken WVE-N531 in an earlier study. It continues to check the medicine's safety and effects over a longer period.
This study tests a new medication called KER-065 to see if it helps people with Duchenne Muscular Dystrophy (DMD). It may help boys and men who are still walking, as well as those who use a wheelchair, as long as they are on a stable corticosteroid treatment.
This study follows boys with Duchenne muscular dystrophy over time to better understand how the condition changes and how standard treatments affect health and function. It does not appear to test a new medicine, but it may help doctors plan better future care and studies.
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