Last updated on February 2020

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi


Brief description of study

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) Type 1, Type 2 or Type 3 who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy (developmental milestones) and durability of response to onasemnogene abeparvovec-xioi treatment.

Clinical Study Identifier: NCT04042025

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