A National Spinal Muscular Atrophy Registry for Real-World Evidence.
Victoria L HodgkinsonMaryam OskouiJoshua LounsberrySaïd M'DahomaEmily ButlerCraig CampbellAlex MacKenzieHugh J McmillanLouise SimardJiri VajsarBernard BraisKristine M ChapmanNicolas ChrestianMeghan CronePeter DobrowolskiSusan DojeijiJames J DowlingNicolas DupreAngela GengeHernan GonorazkySimona HasalAaron IzenbergWendy JohnstonEdward LeungHanns LochmüllerJean K MahAlier MarerroRami MassieLaura McAdamAnna McCormickMichel MelansonMichelle M MezeiCam-Tu E NguyenColleen O'ConnellErin K O'FerrallGerald PfefferCecile PhanStephanie PlamondonChantal PoulinXavier RodrigueKerri L SchellenbergKathy SelbyJordan SherikoChristen ShoesmithGarth SmithMonique TaillonSean TaylorJodi Warman ChardonScott WorleyLawrence KorngutPublished in: The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques (2020)
Prospective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients.