Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.
Arlene M D'SilvaSandra HollandDidu S T KariyawasamKaren HerbertPeter BarclayAnita CairnsSuzanna C MacLennanMonique M RyanHugo SampaioNicholas SmithIan R WoodcockEppie M YiuIan E AlexanderMichelle A FarrarPublished in: Annals of clinical and translational neurology (2022)
This study provides real-world evidence to inform treatment decisions and guide therapeutic expectations for onasemnogene abeparvovec and combination therapy for SMA in health practice, especially for children weighing ≥8 kg receiving higher vector loads. Proactive clinical and laboratory surveillance is essential to facilitate individualised management of risks.