Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Long-term follow-up of AVXS-101 (Zolgensma) in patients with spinal muscular atrophy
2024-513086-39-00Phase 3Authorised
- Sponsor
- Novartis
- Start
- Jun 2024
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 9
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoints: Number of participants who reach developmental milestones, Change from baseline in HFMSE score and 18 more. Planned start Jun 2024; status: authorised.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
Beyond the preview
Go deeper on 2024-513086-39-00.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2024-513086-39-00.