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.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Number of Participants Who Reach Developmental Milestones
Timeframe: Up to 5 years
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
Timeframe: Up to 15 years
Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
Timeframe: Up to 5 years
Change From Baseline in Height Measurements
Timeframe: Up to 5 years
Change From Baseline in Weight Measurements
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
Timeframe: Year 6 to Year 15
Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
Timeframe: Up to 15 years
Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
Timeframe: Up to 15 years
Change From Baseline in Bayley Scales of Infant and Toddler Development
Timeframe: Up to 42 months, 15 days of age
Change From Baseline in Revised Upper Limb Module (RULM) Score
Timeframe: Up to 5 years
Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
Timeframe: Up to 5 years
Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
Timeframe: Up to 5 years
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
Timeframe: Up to 5 years
Number of Participants With Concomitant Medications Overall and by Type of Medications
Timeframe: Up to 5 years
Number of Participants With Other SMA Therapies Overall and by Type of Medications
Timeframe: Year 6 to Year 15