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 Shell

Published in: Pediatric pulmonology (2018)

ClinicalTrials.gov number, NCT02122952.

Keyphrases

replacement therapy
smoking cessation
copy number
genome wide
genome wide identification