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9. The new horizons for treatment of Late-Onset Pompe Disease (LOPD). Guémy C; Laforêt P Rev Neurol (Paris); 2023; 179(1-2):81-89. PubMed ID: 36609019 [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]
11. Response to Comments on "Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice". George K; Anding A J Pharmacol Exp Ther; 2024 May; 389(3):313-314. PubMed ID: 38772716 [TBL] [Abstract][Full Text] [Related]
12. Avalglucosidase alfa: First Approval. Dhillon S Drugs; 2021 Oct; 81(15):1803-1809. PubMed ID: 34591286 [TBL] [Abstract][Full Text] [Related]
13. Improved efficacy of a next-generation ERT in murine Pompe disease. Xu S; Lun Y; Frascella M; Garcia A; Soska R; Nair A; Ponery AS; Schilling A; Feng J; Tuske S; Valle MCD; Martina JA; Ralston E; Gotschall R; Valenzano KJ; Puertollano R; Do HV; Raben N; Khanna R JCI Insight; 2019 Mar; 4(5):. PubMed ID: 30843882 [TBL] [Abstract][Full Text] [Related]
14. 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]
15. 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]
16. 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]
17. 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]
18. New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond. Kishnani PS; Beckemeyer AA Pediatr Endocrinol Rev; 2014 Sep; 12 Suppl 1():114-24. PubMed ID: 25345093 [TBL] [Abstract][Full Text] [Related]
19. 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]
20. Transcriptomic characterization of clinical skeletal muscle biopsy from late-onset Pompe patients. Kinton S; Dufault MR; Zhang M; George K Mol Genet Metab; 2023 Mar; 138(3):107526. PubMed ID: 36774918 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]