This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) 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 and durability of response to onasemnogene abeparvovec-xioi treatment.
ACTIVE_NOT_RECRUITING
Inclusion Criteria:
* Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
* Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Exclusion Criteria:
* Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
PHASE3
ALL
N/A
N/A
2025-04-25
N/A
BIOLOGICAL
Onasemnogene Abeparvovec-xioi
For more information and to contact the study team: