Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Schoser B.Roberts M.Byrne B.J.Sitaraman S.Jiang H.Lafor?t P.Toscano A.Castelli J.D?az-Manera J.Goldman M.van der Ploeg A.T.Bratkovic D.Kuchipudi S.Mozaffar T.Kishnani P.S.Sebok A.Pestronk A.Dominovic-Kovacevic A.Khan A.Koritnik B.Tard C.Lindberg C.Quinn C.Eldridge C.Bodkin C.Reyes-Leiva D.Hughes D.Stefanescu E.SALORT-CAMPANA E.Butler E.Bouhour F.Kim G.Konstantinos Papadimas G.Parenti G.Bartosik-Psujek H.Kushlaf H.Akihiro H.Lau H.Pedro H.Andersen H.Amartino H.Shiraishi H.Kobayashi H.Tarnev I.Vengoechea J.Avelar J.Shin J.-H.Cauci J.Alonso-P?rez J.Janszky J.Berthy J.Cornelia K.Gutschmidt K.Claeys K.Judit Molnar M.Wencel M.Tarnopolsky M.Dimachkie M.Tchan M.Freimer M.Longo N.Vidal-Fernandez N.Musumeci O.Goker-Alpan O.Deegan P.Clemens P.R.Roxburgh R.Henderson R.Hopkin R.Sacconi S.Fecarotta S.Attarian S.Wenninger S.Dearmey S.Hiwot T.Burrow T.Ruck T.Sawada T.Laszlo V.L?scher W.YIN-HSIU CHIENPROPEL Study Group2022-03-282022-03-2820211474-4422https://www.scopus.com/inward/record.uri?eid=2-s2.0-85119194577&doi=10.1016%2fS1474-4422%2821%2900331-8&partnerID=40&md5=5b6e810d3470dfcbb2e5d83bff96c54ehttps://scholars.lib.ntu.edu.tw/handle/123456789/602263[SDGs]SDG3Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trialjournal article10.1016/S1474-4422(21)00331-8348004002-s2.0-85119194577