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

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 onasemnogene...

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Detalles Bibliográficos
Autores principales:	D'Silva, Arlene M., Holland, Sandra, Kariyawasam, Didu, Herbert, Karen, Barclay, Peter, Cairns, Anita, MacLennan, Suzanna C., Ryan, Monique M., Sampaio, Hugo, Smith, Nicholas, Woodcock, Ian R., Yiu, Eppie M., Alexander, Ian E., Farrar, Michelle A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2022
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8935277/ https://www.ncbi.nlm.nih.gov/pubmed/35170254 http://dx.doi.org/10.1002/acn3.51519

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