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Pediatric Pulmonology, ISSN 8755-6863, 02/2019, Volume 54, Issue 2, pp. 179 - 185
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... 
health outcomes | gene replacement | AVXS‐101 | gene therapy | spinal muscular atrophy | quality of life | SMA1 | AVXS-101 | DIAGNOSIS | MANAGEMENT | MODEL | CARE | CHILDREN | DELIVERY | RESPIRATORY SYSTEM | PEDIATRICS | QUALITY-OF-LIFE | NONINVASIVE VENTILATION | Original | Pcd, Pig, Nehi, Child, and Rare Diseases
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