AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

This study is looking for boys who have Duchenne muscular dystrophy. They need to be male and at least 1 year old, but less than 12 years old. They should also meet a certain weight requirement (at least 10 kg).

To be considered, participants generally need to have specific changes in their DMD gene and may need to be able to walk a certain distance independently. They should also have been on a stable dose of steroid medication (a common treatment for DMD) for at least 12 weeks. Your doctor will check your child's general health, including their liver and kidney function, to make sure they are healthy enough to take part.

It's important that the child's parent or legal guardian understands the study and agrees to participate, and that they can bring their child to all scheduled appointments and follow the study instructions.

If you join this study, your child will receive the RGX-202 treatment once, through a drip into their vein. Before and during the treatment, your child will also take other medicines for a short time to help prevent any strong reactions to the gene therapy.

The study involves regular visits to the clinic over roughly two years to check your child's health, how they are tolerating the treatment, and if their muscles are improving. These visits will include various assessments, such as physical examinations, blood tests, and tests to measure muscle strength and function. After the main study period, there might be an option to join a separate long-term follow-up study to continue monitoring your child's health.