Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA) (2301)

R. Mendell, Jerry; S. Finkel, Richard; Mercuri, Eugenio; A. Strauss, Kevin; Day, John; de Kleyn, A.; H. Chand, Deepa; Tauscher‐Wisniewski, Sitra; N. Meriggioli, Matthew

doi:10.1212/wnl.96.15_supplement.2301

Public

Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA) (2301)

Published in Neurology • Apr 13, 2021

Authors:

Jerry R. Mendell

Richard S. Finkel

Eugenio Mercuri

Abstract

Evaluate long-term safety/efficacy of patients previously treated in the onasemnogene abeparvovec (formally AVXS-101) clinical development program who electively enrolled into LTFU studies LT-001 (NCT03421977) and LT-002 (NCT04042025).

Subject

SMA*

Spinal muscular atrophy

Medicine

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