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ENROLLING BY INVITATION
NCT06588803
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
Sponsor: Guangzhou Jiayin Biotech Ltd
View on ClinicalTrials.gov
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)
Official title: A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy (SMA)
Key Details
Gender
All
Age Range
1 Month - Any
Study Type
OBSERVATIONAL
Enrollment
18
Start Date
2025-03
Completion Date
2030-12-31
Last Updated
2025-03-05
Healthy Volunteers
No
Conditions
Interventions
OTHER
No intervention, only for observational studies
No intervention, only for observational studies
Locations (1)
The Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China