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AAN 2021 | START trial long-term follow-up of onasemnogene abeparvovec in SMA

Jerry Mendell • 6 Apr 2021
AAN 2021
Neuromuscular Disorders Spinal Muscular Atrophy Onasemnogene abeparvovec

Jerry Mendell, MD, FAAN, Nationwide Children’s Hospital & Ohio State University, Columbus, OH, discusses the findings of the START trial (NCT02122952) long-term follow-up of onasemnogene abeparvovec gene therapy in spinal muscular atrophy (SMA). This trial analyzed patients that received a single infusion of gene sequence between 2014 and 2017. The results validate the expected goals showing a consistent improvement in patients’ symptoms. This interview took place during the American Academy of Neurology (AAN) 2021 Annual Meeting.

Disclosures

Prof. Mendell reports the following disclosures:
Consultant for Sarepta therapeutics, Novartis Inc, Vertex Inc

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