Gene therapy for young boys with Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests a one-time gene therapy given by injection into the spinal fluid (intrathecal) for boys with Duchenne muscular dystrophy. It aims to see if the treatment is safe and how it spreads in the body, and may offer a new way to slow the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be a boy between 2 and 5 years old.
- He must be able to walk at least 10 meters (about 33 feet) without help or assistive devices.
- A genetic test must confirm a specific type of Duchenne mutation (between exons 18 and 58).
- He cannot have had prior gene or cell therapy, or certain other DMD treatments in the last 6 months.
- He must be up to date on recommended childhood vaccines (except flu and COVID-19 vaccines, which are optional).
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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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 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 Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
This trial tests a gene therapy called GNR-097 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help improve muscle function, and the study will check how safe and effective it is.
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.
This trial tests a new cell-based treatment for Duchenne muscular dystrophy in adults who can no longer walk. The goal is to see if the therapy can help improve muscle function.
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