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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD): The ENVISION Trial

Research Question:
Is the gene therapy, called delandistrogene moxeparvovec, safe and effective at treating Duchenne Muscular Dystrophy (DMD)?

Basic Study Information

DMD is caused by a non-working gene that causes an important protein called dystrophin to not be made properly. Dystrophin helps protect the muscles from breaking down and becoming weaker. This study will help researchers find out if replacing the non-working gene with a new one is safe and effective at treating DMD. This study will be done in two parts and all participants will have the opportunity to receive the therapy in either Part 1 or Part 2. Patients are eligible if they are are male at birth, have a diagnosis of DMD and have the genetic mutation that leads to the complete absence of the dystrophin protein.

Location: University of Rochester, Department of Neurology
Study Reference #: STUDY00007767

Lead Researcher (Principal Investigator)

Lead Researcher:  Emma Ciafaloni

Study Contact Information

Study Coordinator: Dr. Emma Ciafaloni
Phone: (585) 275-4568

Additional Study Details

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