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
Purpose:
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
Email: Emma_Ciafaloni@URMC.Rochester.edu
Additional Study Details
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