Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

Abstract Objective To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA). Methods A prospective cohort study of children with SMA treated with onasem...

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Bibliographic Details
Main Authors: Arlene M. D'Silva, Sandra Holland, Didu Kariyawasam, Karen Herbert, Peter Barclay, Anita Cairns, Suzanna C. MacLennan, Monique M. Ryan, Hugo Sampaio, Nicholas Smith, Ian R. Woodcock, Eppie M. Yiu, Ian E. Alexander, Michelle A. Farrar
Format: Article
Language:English
Published: Wiley 2022-03-01
Series:Annals of Clinical and Translational Neurology
Online Access:https://doi.org/10.1002/acn3.51519

Internet

https://doi.org/10.1002/acn3.51519

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