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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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