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...

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Detaylı Bibliyografya
Yayımlandı:Pediatr Pulmonol
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: John Wiley and Sons Inc. 2018
Konular:
Original Articles
Online Erişim: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
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