Study Summary
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
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AVXS-101BIOLOGICAL
Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | United States |