Trial Search Results
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
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.
Stanford is currently not accepting patients for this trial.
Novartis Gene Therapies
- Biological: Onasemnogene Abeparvovec-xioi
- 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
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up
Ages Eligible for Study
N/A - N/A
Genders Eligible for Study