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3. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Diaz-Manera J; Kishnani PS; Kushlaf H; Ladha S; Mozaffar T; Straub V; Toscano A; van der Ploeg AT; Berger KI; Clemens PR; Chien YH; Day JW; Illarioshkin S; Roberts M; Attarian S; Borges JL; Bouhour F; Choi YC; Erdem-Ozdamar S; Goker-Alpan O; Kostera-Pruszczyk A; Haack KA; Hug C; Huynh-Ba O; Johnson J; Thibault N; Zhou T; Dimachkie MM; Schoser B; Lancet Neurol; 2021 Dec; 20(12):1012-1026. PubMed ID: 34800399 [TBL] [Abstract][Full Text] [Related]
4. 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07). Schoser B; Kishnani PS; Bratkovic D; Byrne BJ; Claeys KG; Díaz-Manera J; Laforêt P; Roberts M; Toscano A; van der Ploeg AT; Castelli J; Goldman M; Holdbrook F; Sitaraman Das S; Wasfi Y; Mozaffar T; J Neurol; 2024 May; 271(5):2810-2823. PubMed ID: 38418563 [TBL] [Abstract][Full Text] [Related]
5. Enzyme replacement therapy for infantile-onset Pompe disease. Chen M; Zhang L; Quan S Cochrane Database Syst Rev; 2017 Nov; 11(11):CD011539. PubMed ID: 29155436 [TBL] [Abstract][Full Text] [Related]
6. Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data. Shohet S; Hummel N; Fu S; Keyzor I; MacCulloch A; Johnson N; Castelli J; Czarny-Ozga I; Mozaffar T; Thom H J Comp Eff Res; 2024 Oct; 13(10):e240045. PubMed ID: 39287071 [No Abstract] [Full Text] [Related]
7. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease. Dimachkie MM; Barohn RJ; Byrne B; Goker-Alpan O; Kishnani PS; Ladha S; Laforêt P; Mengel KE; Peña LDM; Sacconi S; Straub V; Trivedi J; Van Damme P; van der Ploeg AT; Vissing J; Young P; Haack KA; Foster M; Gilbert JM; Miossec P; Vitse O; Zhou T; Schoser B; Neurology; 2022 Aug; 99(5):e536-e548. PubMed ID: 35618441 [TBL] [Abstract][Full Text] [Related]
8. Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease. Fiege L; Duran I; Marquardt T Pharmaceuticals (Basel); 2023 Aug; 16(9):. PubMed ID: 37765007 [TBL] [Abstract][Full Text] [Related]
9. Cipaglucosidase Alfa: First Approval. Blair HA Drugs; 2023 Jun; 83(8):739-745. PubMed ID: 37184753 [TBL] [Abstract][Full Text] [Related]
10. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study. Pena LDM; Barohn RJ; Byrne BJ; Desnuelle C; Goker-Alpan O; Ladha S; Laforêt P; Mengel KE; Pestronk A; Pouget J; Schoser B; Straub V; Trivedi J; Van Damme P; Vissing J; Young P; Kacena K; Shafi R; Thurberg BL; Culm-Merdek K; van der Ploeg AT; Neuromuscul Disord; 2019 Mar; 29(3):167-186. PubMed ID: 30770310 [TBL] [Abstract][Full Text] [Related]
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12. Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice. Anding A; Kinton S; Baranowski K; Brezzani A; De Busser H; Dufault MR; Finn P; Keefe K; Tetrault T; Li Y; Qiu W; Raes K; Vitse O; Zhang M; Ziegler R; Sardi SP; Hunter B; George K J Pharmacol Exp Ther; 2023 Nov; 387(2):188-203. PubMed ID: 37679046 [TBL] [Abstract][Full Text] [Related]
13. Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial. MacCulloch A; Griffiths A; Johnson N; Shohet S J Health Econ Outcomes Res; 2024; 11(2):80-85. PubMed ID: 39318718 [No Abstract] [Full Text] [Related]
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18. Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial. Toscano A; Pollissard L; Msihid J; van der Beek N; Kishnani PS; Dimachkie MM; Berger KI; DasMahapatra P; Thibault N; Hamed A; Zhou T; Haack KA; Schoser B Mol Genet Metab; 2024 Feb; 141(2):108121. PubMed ID: 38184428 [TBL] [Abstract][Full Text] [Related]
19. A randomized study of alglucosidase alfa in late-onset Pompe's disease. van der Ploeg AT; Clemens PR; Corzo D; Escolar DM; Florence J; Groeneveld GJ; Herson S; Kishnani PS; Laforet P; Lake SL; Lange DJ; Leshner RT; Mayhew JE; Morgan C; Nozaki K; Park DJ; Pestronk A; Rosenbloom B; Skrinar A; van Capelle CI; van der Beek NA; Wasserstein M; Zivkovic SA N Engl J Med; 2010 Apr; 362(15):1396-406. PubMed ID: 20393176 [TBL] [Abstract][Full Text] [Related]
20. Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial. Strothotte S; Strigl-Pill N; Grunert B; Kornblum C; Eger K; Wessig C; Deschauer M; Breunig F; Glocker FX; Vielhaber S; Brejova A; Hilz M; Reiners K; Müller-Felber W; Mengel E; Spranger M; Schoser B J Neurol; 2010 Jan; 257(1):91-7. PubMed ID: 19649685 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]