Home > Neurology > AAN 2022 > Muscle and Neuro-Muscular Disorders > Gene therapy effective in older patients with spinal muscular atrophy

Gene therapy effective in older patients with spinal muscular atrophy

Presented By
Prof. Laurent Servais, MDUK Oxford Neuromuscular Centre, UK
Conference
AAN 2022
Trial
RESTORE
Doi
https://doi.org/10.55788/cf89271f
The observational RESTORE study demonstrated efficacy of onasemnogene abeparvovec (OA) in patients with spinal muscular atrophy (SMA) aged 6 months or older at the time they received this gene therapy. Interventional trials of OA had already demonstrated safety and efficacy of OA in patients <6 months of age. RESTORE (NCT04174157) is a comprehensive, prospective, multicentre, observational registry of patients with genetically-confirmed SMA across several countries. At the time of OA infusion, 117 patients were 6 months or older. Of these, 51 were 6–12 months old, 57 were 12–24 months, and 9 were 24 months or older. Most participants (n=73) had 2 copies of the SMN2 gene; one third (n=38) received OA monotherapy, while 26 combined OA with nusinersen, risdiplam, or both, and 53 patients switched from nusinersen to OA. Prof. Laurent Servais (MDUK...


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