Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
Sign Up to like & getrecommendations! Published in 2019 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.24203
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… read more here.
Keywords: health outcomes; spinal muscular; avxs 101; atrophy type ... See more keywords