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Neurology

CGTLive’s Weekly Rewind – September 30, 2022

Review top news and interview highlights from the week ending September 30, 2022.

Working With the Community to Improve Patient Access to Trials, Therapies

Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.

Sarepta’s DMD Gene Therapy Goes In Front of FDA With BLA Submission

SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.