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8. Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation. Desai AK; Walters CK; Cope HL; Kazi ZB; DeArmey SM; Kishnani PS Mol Genet Metab; 2018 Feb; 123(2):92-96. PubMed ID: 29289479 [TBL] [Abstract][Full Text] [Related]
9. Characteristics of Pompe disease in China: a report from the Pompe registry. Zhao Y; Wang Z; Lu J; Gu X; Huang Y; Qiu Z; Wei Y; Yan C Orphanet J Rare Dis; 2019 Apr; 14(1):78. PubMed ID: 30943998 [TBL] [Abstract][Full Text] [Related]
10. Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study. van der Ploeg A; Carlier PG; Carlier RY; Kissel JT; Schoser B; Wenninger S; Pestronk A; Barohn RJ; Dimachkie MM; Goker-Alpan O; Mozaffar T; Pena LD; Simmons Z; Straub V; Guglieri M; Young P; Boentert M; Baudin PY; Wens S; Shafi R; Bjartmar C; Thurberg BL Mol Genet Metab; 2016 Sep; 119(1-2):115-23. PubMed ID: 27473031 [TBL] [Abstract][Full Text] [Related]
11. Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD). Ripolone M; Violano R; Ronchi D; Mondello S; Nascimbeni A; Colombo I; Fagiolari G; Bordoni A; Fortunato F; Lucchini V; Saredi S; Filosto M; Musumeci O; Tonin P; Mongini T; Previtali S; Morandi L; Angelini C; Mora M; Sandri M; Sciacco M; Toscano A; Comi GP; Moggio M Neuropathol Appl Neurobiol; 2018 Aug; 44(5):449-462. PubMed ID: 28574618 [TBL] [Abstract][Full Text] [Related]
12. Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease. Hundsberger T; Rohrbach M; Kern L; Rösler KM J Neurol; 2013 Sep; 260(9):2279-85. PubMed ID: 23749294 [TBL] [Abstract][Full Text] [Related]
13. Adjunctive albuterol enhances the response to enzyme replacement therapy in late-onset Pompe disease. Koeberl DD; Austin S; Case LE; Smith EC; Buckley AF; Young SP; Bali D; Kishnani PS FASEB J; 2014 May; 28(5):2171-6. PubMed ID: 24443373 [TBL] [Abstract][Full Text] [Related]
14. 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]
15. Use of cardiac magnetic resonance imaging to evaluate cardiac structure, function and fibrosis in children with infantile Pompe disease on enzyme replacement therapy. Barker PC; Pasquali SK; Darty S; Ing RJ; Li JS; Kim RJ; DeArmey S; Kishnani PS; Campbell MJ Mol Genet Metab; 2010 Dec; 101(4):332-7. PubMed ID: 20875764 [TBL] [Abstract][Full Text] [Related]
16. [Pompe disease treated with enzyme replacement therapy in pregnancy]. Grosz Z; Várdi KV; Molnár JM Ideggyogy Sz; 2020 Sep; 73(9-10):339-344. PubMed ID: 33035415 [TBL] [Abstract][Full Text] [Related]
17. Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start. Stockton DW; Kishnani P; van der Ploeg A; Llerena J; Boentert M; Roberts M; Byrne BJ; Araujo R; Maruti SS; Thibault N; Verhulst K; Berger KI J Neurol; 2020 Oct; 267(10):3038-3053. PubMed ID: 32524257 [TBL] [Abstract][Full Text] [Related]
18. Effects of enzyme replacement therapy on bone density in late onset Pompe disease. Avanti M; Martin A; Columbres RC; Mozaffar T; Kimonis V Mol Genet Metab; 2023 Nov; 140(3):107644. PubMed ID: 37515933 [TBL] [Abstract][Full Text] [Related]
19. Quantitative computed tomography for enzyme replacement therapy in Pompe disease. Yonee C; Toyoshima M; Young SP; Maruyama S; Higuchi I; Narita A; Maegaki Y; Nanba E; Ohno K; Kawano Y Brain Dev; 2012 Nov; 34(10):834-9. PubMed ID: 22521436 [TBL] [Abstract][Full Text] [Related]