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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...
Autores principales: | , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6590370/ https://www.ncbi.nlm.nih.gov/pubmed/30548438 http://dx.doi.org/10.1002/ppul.24203 |