Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2 (4163)

Kevin A. Strauss; Francesco Muntoni; Michelle A. Farrar; Kayoko Saito; Jerry R. Mendell; Laurent Servais; Hugh J. McMillan; Kathryn J. Swoboda; Jennifer M. Kwon; Craig M. Zaidman; Claudia A. Chiriboga; Susan T. Iannaccone; Jena M. Krueger; Julie Parsons; Perry B. Shieh; Sarah Kavanagh; Deepa H. Chand; Sitra Tauscher‐Wisniewski; Thomas A. Macek

doi:10.1212/wnl.96.15_supplement.4163

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Abstract

Evaluate the safety/efficacy of onasemnogene abeparvovec (formerly AVXS-101) in presymptomatic SMA patients with 3 copies of the survival motor neuron 2 gene (SMN2).

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Strauss et al. (2021) studied this question.

synapsesocial.com/papers/6a95720aaa92d0f3572ca3ce https://doi.org/10.1212/wnl.96.15_supplement.4163

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Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2 (4163)

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Abstract

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Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2 (4163)

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