No intervention, only for observational studies: No intervention, only for observational studies
Study summary
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
Eligibility
Sex
ALL
Min age
1 Month
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Previously received treatment with EXG001-307 in trial EXG001-307-102;
2. Parents or guardians understand the research procedure and sign the ICF; Good compliance and willingness to follow research procedures. Voluntarily participate in this clinical trial.
Exclusion Criteria:
1\. Parents or guardians are unwilling or unable to participate in long-term follow-up studies.
Primary outcome measure(s)
long-term safety：Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs) — 4 years Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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