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

Join a study on new gene therapy for Duchenne Muscular Dystrophy!

Recruiting
1 years and older
Male
Phase 2/3
  • Simplified description (AI rewritten)
  • Technical description (Original)
RGX-202 Gene Therapy Study for Duchenne Muscular Dystrophy (DMD) RGX-202 is an experimental gene therapy aiming to help people with Duchenne Muscular Dystrophy (DMD), a rare condition caused by problems in the gene that makes dystrophin, a vital protein for muscle strength. Without this protein, muscles can get damaged and weaken over time. The study tests a one-time dose of RGX-202, which uses a special tool called the AAV8 vector to deliver tiny versions of the dystrophin gene to muscle cells. Key Points:
  • The study lasts for 104 weeks with visits at different times to assess safety and effects.
  • Participants must be boys aged 1 to under 12 years and able to meet specific walking criteria.
  • Participants will follow a detailed treatment plan, including short-term drugs to manage immune responses.
The study has three parts, each with different goals and participant numbers. To join, boys need to meet certain health criteria and their guardians must agree to the study terms. More information is available through a provided link.
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Study details
    Duchenne Muscular Dystrophy

NCT05693142

REGENXBIO Inc.

7 March 2026

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