The Potential of Gene Transfer Therapy in SMA
John Brandsema, MD, discusses gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.
A Rational Approach to Assessing SMA Therapies: Julie Parsons, MD
The primary investigator of the phase 4 RESPOND study discussed the role that postmarketing studies can play in assessing treatments for SMA.
Phase 4 RESPOND Trial and the Need for Similar Trials in SMA: Julie Parsons, MD
The primary investigator of RESPOND discussed the trial’s goals and measures, as well as insight into why clinical trials like it are important in spinal muscular atrophy.
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