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3. Ptosis, extraocular motility disorder, and myopia as features of pompe disease. Slingerland NW; Polling JR; van Gelder CM; van der Ploeg AT; Bleyen I Orbit; 2011 Mar; 30(2):111-3. PubMed ID: 21438734 [TBL] [Abstract][Full Text] [Related]
4. Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium. Ditters IAM; Huidekoper HH; Kruijshaar ME; Rizopoulos D; Hahn A; Mongini TE; Labarthe F; Tardieu M; Chabrol B; Brassier A; Parini R; Parenti G; van der Beek NAME; van der Ploeg AT; van den Hout JMP; Lancet Child Adolesc Health; 2022 Jan; 6(1):28-37. PubMed ID: 34822769 [TBL] [Abstract][Full Text] [Related]
5. 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]
6. A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease. Han SO; Pope R; Li S; Kishnani PS; Steet R; Koeberl DD Mol Genet Metab; 2016 Feb; 117(2):114-9. PubMed ID: 26454691 [TBL] [Abstract][Full Text] [Related]
7. 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]
8. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease. Case LE; Bjartmar C; Morgan C; Casey R; Charrow J; Clancy JP; Dasouki M; DeArmey S; Nedd K; Nevins M; Peters H; Phillips D; Spigelman Z; Tifft C; Kishnani PS Neuromuscul Disord; 2015 Apr; 25(4):321-32. PubMed ID: 25617983 [TBL] [Abstract][Full Text] [Related]
9. Cognitive outcome of patients with classic infantile Pompe disease receiving enzyme therapy. Ebbink BJ; Aarsen FK; van Gelder CM; van den Hout JM; Weisglas-Kuperus N; Jaeken J; Lequin MH; Arts WF; van der Ploeg AT Neurology; 2012 May; 78(19):1512-8. PubMed ID: 22539577 [TBL] [Abstract][Full Text] [Related]
11. Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease. van Gelder CM; Hoogeveen-Westerveld M; Kroos MA; Plug I; van der Ploeg AT; Reuser AJ J Inherit Metab Dis; 2015 Mar; 38(2):305-14. PubMed ID: 24715333 [TBL] [Abstract][Full Text] [Related]
12. Quantification of muscle pathology in infantile Pompe disease. Schänzer A; Kaiser AK; Mühlfeld C; Kulessa M; Paulus W; von Pein H; Rohrbach M; Viergutz L; Mengel E; Marquardt T; Neubauer B; Acker T; Hahn A Neuromuscul Disord; 2017 Feb; 27(2):141-152. PubMed ID: 27927596 [TBL] [Abstract][Full Text] [Related]
13. 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]
14. Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease. Kanters TA; Hoogenboom-Plug I; Rutten-Van Mölken MP; Redekop WK; van der Ploeg AT; Hakkaart L Orphanet J Rare Dis; 2014 May; 9():75. PubMed ID: 24884717 [TBL] [Abstract][Full Text] [Related]
15. [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]
16. Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy. van der Meijden JC; Kruijshaar ME; Harlaar L; Rizopoulos D; van der Beek NAME; van der Ploeg AT J Inherit Metab Dis; 2018 Nov; 41(6):1205-1214. PubMed ID: 29556838 [TBL] [Abstract][Full Text] [Related]
17. The cost-effectiveness of enzyme replacement therapy (ERT) for the infantile form of Pompe disease: comparing a high-income country's approach (England) to that of a middle-income one (Colombia). Castro-Jaramillo HE Rev Salud Publica (Bogota); 2012; 14(1):143-55. PubMed ID: 23250322 [TBL] [Abstract][Full Text] [Related]