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2 clinical studies listed.
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Tundra lists 2 Spinal Muscular Atrophy 1 clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT06191354
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
Gender: All
Ages: Any - 180 Days
Updated: 2025-11-21
1 state
NCT03421977
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
Gender: All
Updated: 2025-04-25
1 state