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 Farrar

Published 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.

Keyphrases

public health
healthcare
primary care
young adults
human health
mental health
risk assessment
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