Study Summary
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
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Single dose intravenous of BBM-D101GENETIC
BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.The administration is completed by a single intravenous infusion.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | Beijing Municipality | China |