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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… Click to show full abstract
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.
Keywords:
health outcomes;
spinal muscular;
avxs 101;
atrophy type;
muscular atrophy;
101 gene
Journal Title: Pediatric Pulmonology
Year Published: 2019
Link to full text (if available)
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Journal Title: Pediatric Pulmonology
Year Published: 2019
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!