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6. 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]
7. Prognostic factors for the late onset Pompe disease with enzyme replacement therapy: from our experience of 4 cases including an autopsy case. Kobayashi H; Shimada Y; Ikegami M; Kawai T; Sakurai K; Urashima T; Ijima M; Fujiwara M; Kaneshiro E; Ohashi T; Eto Y; Ishigaki K; Osawa M; Kyosen SO; Ida H Mol Genet Metab; 2010 May; 100(1):14-9. PubMed ID: 20202878 [TBL] [Abstract][Full Text] [Related]
8. High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa. de Vries JM; van der Beek NA; Kroos MA; Ozkan L; van Doorn PA; Richards SM; Sung CC; Brugma JD; Zandbergen AA; van der Ploeg AT; Reuser AJ Mol Genet Metab; 2010 Dec; 101(4):338-45. PubMed ID: 20826098 [TBL] [Abstract][Full Text] [Related]
9. Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease. Nilsson MI; Samjoo IA; Hettinga BP; Koeberl DD; Zhang H; Hawke TJ; Nissar AA; Ali T; Brandt L; Ansari MU; Hazari H; Patel N; Amon J; Tarnopolsky MA Mol Genet Metab; 2012 Nov; 107(3):469-79. PubMed ID: 23041258 [TBL] [Abstract][Full Text] [Related]
10. Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa. Gragnaniello V; Fecarotta S; Pecoraro A; Tarallo A; Catzola A; Spadaro G; Parenti G; Della Casa R Neurol Sci; 2019 Jul; 40(7):1453-1455. PubMed ID: 30778879 [TBL] [Abstract][Full Text] [Related]
12. Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease. Papadopoulos C; Orlikowski D; Prigent H; Lacour A; Tard C; Furby A; Praline J; Solé G; Hogrel JY; De Antonio M; Semplicini C; Deibener-Kaminsky J; Kaminsky P; Eymard B; Taouagh N; Perniconi B; Hamroun D; Laforêt P; Mol Genet Metab; 2017 Sep; 122(1-2):80-85. PubMed ID: 28648663 [TBL] [Abstract][Full Text] [Related]
13. The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease. Lacaná E; Yao LP; Pariser AR; Rosenberg AS Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):30-9. PubMed ID: 22253234 [TBL] [Abstract][Full Text] [Related]
14. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. van der Ploeg AT; Barohn R; Carlson L; Charrow J; Clemens PR; Hopkin RJ; Kishnani PS; Laforêt P; Morgan C; Nations S; Pestronk A; Plotkin H; Rosenbloom BE; Sims KB; Tsao E Mol Genet Metab; 2012 Nov; 107(3):456-61. PubMed ID: 23031366 [TBL] [Abstract][Full Text] [Related]
15. The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management. Kishnani PS; Beckemeyer AA; Mendelsohn NJ Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):1-7. PubMed ID: 22253049 [TBL] [Abstract][Full Text] [Related]
16. Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month follow-up study. Klinge L; Straub V; Neudorf U; Voit T Neuropediatrics; 2005 Feb; 36(1):6-11. PubMed ID: 15776317 [TBL] [Abstract][Full Text] [Related]
17. Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations. Case LE; Beckemeyer AA; Kishnani PS Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):69-79. PubMed ID: 22252989 [TBL] [Abstract][Full Text] [Related]
18. Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry. Semplicini C; De Antonio M; Taouagh N; Béhin A; Bouhour F; Echaniz-Laguna A; Magot A; Nadaj-Pakleza A; Orlikowski D; Sacconi S; Salort-Campana E; Solé G; Tard C; Zagnoli F; Hogrel JY; Hamroun D; Laforêt P; J Inherit Metab Dis; 2020 Nov; 43(6):1219-1231. PubMed ID: 32515844 [TBL] [Abstract][Full Text] [Related]
19. Alglucosidase alfa therapy for Pompe disease in pregnancy - Case report. Kłos J; Kwaśniak-Butowska M; Sławek J J Neurol Sci; 2017 Apr; 375():167-169. PubMed ID: 28320122 [No Abstract] [Full Text] [Related]