Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

BACKGROUND: Spinal Muscular Atrophy type 1 (SMA1) is a rare genetic neuromuscular disease where 75% of SMA1 patients die/require permanent‐ventilation by 13.6 months. This study assessed the health outcomes of SMA1 infants treated with AVXS‐101 gene replacement therapy. METHODS: Twelve genetically c...

Descripción completa

Guardado en:
Detalles Bibliográficos
Publicado en:Pediatr Pulmonol
Autores principales: Al‐Zaidy, Samiah, Pickard, A. Simon, Kotha, Kavitha, Alfano, Lindsay N., Lowes, Linda, Paul, Grace, Church, Kathleen, Lehman, Kelly, Sproule, Douglas M., Dabbous, Omar, Maru, Benit, Berry, Katherine, Arnold, W. David, Kissel, John T., Mendell, Jerry R., Shell, Richard
Formato: Artigo
Lenguaje:Inglês
Publicado: John Wiley and Sons Inc. 2018
Materias:
Original Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6590370/
https://ncbi.nlm.nih.gov/pubmed/30548438
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ppul.24203
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares