Phase 3 Spinal Muscular Atrophy (SMA) Trials
2 Phase 3 trials for Spinal Muscular Atrophy (SMA). Phase 3 trials are the final stage of testing before a treatment can be submitted for FDA approval.
What is a Phase 3 trial?
Phase 3 clinical trials study a treatment in large groups (hundreds to thousands of people) to confirm its effectiveness, monitor side effects, and compare it to standard treatments. These trials are the last major step before the FDA considers approving a new treatment.
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle...
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine...
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial....