Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report

Kishnani, Priya SPriya SKishnaniKronn, DavidDavidKronnBrassier, AnaïsAnaïsBrassierBroomfield, AlexanderAlexanderBroomfieldDavison, JamesJamesDavisonHahn, Si HounSi HounHahnKumada, SatokoSatokoKumadaLabarthe, FrançoisFrançoisLabartheOhki, HirotakaHirotakaOhkiPichard, SamiaSamiaPichardPrakalapakorn, S GraceS GracePrakalapakornHaack, Kristina AnKristina AnHaackKittner, BarbaraBarbaraKittnerMeng, XianzhangXianzhangMengSparks, SusanSusanSparksWilson, CatherineCatherineWilsonZaher, AtefAtefZaherYIN-HSIU CHIEN2023-01-112023-01-112022-12-2010983600https://scholars.lib.ntu.edu.tw/handle/123456789/627122Mini-COMET (NCT03019406; Sanofi) is a phase 2, open-label, ascending-dose, 3-cohort study, evaluating avalglucosidase alfa safety, pharmacokinetics, and efficacy in individuals with infantile-onset Pompe disease aged <18 years who previously received alglucosidase alfa and showed clinical decline (cohorts 1 and 2) or suboptimal response (cohort 3).enAcid α-glucosidase (GAA) deficiency; Alglucosidase alfa; Avalglucosidase alfa; Enzyme replacement therapy; Infantile-onset Pompe disease[SDGs]SDG3Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis reportjournal article10.1016/j.gim.2022.10.010365420862-s2.0-85144503911https://api.elsevier.com/content/abstract/scopus_id/85144503911