Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy - 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; et al. (Pediatric pulmonology, 2019)
Keywords: AVXS‐101; Child, Preschool; gene replacement; gene therapy; Genetic Therapy; health outcomes; Hospitalization; Humans; Infant; Infant, Newborn; Mutation; Neuromuscular diseases; Original; Original : Pcd, Pig, Nehi, Child, and Rare Diseases; quality of life; SMA1; Spinal Muscular Atrophies of Childhood - genetics; Spinal Muscular Atrophies of Childhood - therapy; spinal muscular atrophy; Survival of Motor Neuron 1 Protein - genetics; Treatment Outcome; AVXS-101; health outcomes; gene replacement; gene therapy; spinal muscular atrophy; SMA1; quality of life