onasemnogene abeparvovec
onasemnogene abeparvovec: 4 clinical trials tracked.
onasemnogene abeparvovec evidence translation
What we can confirm
4 tracked trials
What it means
What to watch
Prepared research question
What is verified about onasemnogene abeparvovec, and what remains unknown?
Traceable citations · Unknowns marked
Clinical Trial Registry Records (4)
Onasemnogene Abeparvovec: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Primary endpoint: Change from baseline in HFMSE for independently ambulatory participants
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Primary endpoint: Number of participants with treatment-emergent serious adverse events (SAEs)
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Primary endpoint: Number of Participants Who Reach Developmental Milestones
A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101
Primary endpoint: Long-Term Safety