A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Latest Information Update: 29 Jul 2024
At a glance
- Drugs Onasemnogene-abeparvovec (Primary) ; Onasemnogene-abeparvovec (Primary)
- Indications Spinal muscular atrophy
- Focus Adverse reactions; Therapeutic Use
- Sponsors Novartis Gene Therapies; Novartis Pharmaceuticals
Most Recent Events
- 21 Feb 2024 Planned End Date changed from 29 Dec 2035 to 31 Dec 2035.
- 21 Feb 2024 Planned primary completion date changed from 29 Dec 2035 to 31 Dec 2035.
- 27 Apr 2023 Results (As of may 23, 2022, n=81) assessing term safety and durability of intravenous or intrathecal onasemnogene abeparvovec in symptomatic and presymptomatic patients with spinal muscular atrophy (SMA) who enrolled into the LT-002 study presented at the 75th Annual Meeting of the American Academy of Neurology 2023