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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Detalles Bibliográficos

Principais autores:	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
Formato:	Artigo
Idioma:	Inglês
Publicado:	Wiley 2022-03-01
Series:	Annals of Clinical and Translational Neurology
Acceso en liña:	https://doi.org/10.1002/acn3.51519
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