Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy.
Samiah Al-ZaidyA Simon PickardKavitha KothaLindsay N AlfanoLinda LowesGrace PaulKathleen ChurchKelly LehmanDouglas M SprouleOmar DabbousBenit MaruKatherine BerryW David ArnoldJohn T KisselJerry R MendellRichard ShellPublished in: Pediatric pulmonology (2018)
ClinicalTrials.gov number, NCT02122952.