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2. Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy. Parini R; De Lorenzo P; Dardis A; Burlina A; Cassio A; Cavarzere P; Concolino D; Della Casa R; Deodato F; Donati MA; Fiumara A; Gasperini S; Menni F; Pagliardini V; Sacchini M; Spada M; Taurisano R; Valsecchi MG; Di Rocco M; Bembi B Orphanet J Rare Dis; 2018 Feb; 13(1):32. PubMed ID: 29422078 [TBL] [Abstract][Full Text] [Related]
3. Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease. Messinger YH; Mendelsohn NJ; Rhead W; Dimmock D; Hershkovitz E; Champion M; Jones SA; Olson R; White A; Wells C; Bali D; Case LE; Young SP; Rosenberg AS; Kishnani PS Genet Med; 2012 Jan; 14(1):135-42. PubMed ID: 22237443 [TBL] [Abstract][Full Text] [Related]
10. Novel approaches to quantify CNS involvement in children with Pompe disease. Korlimarla A; Spiridigliozzi GA; Crisp K; Herbert M; Chen S; Malinzak M; Stefanescu M; Austin SL; Cope H; Zimmerman K; Jones H; Provenzale JM; Kishnani PS Neurology; 2020 Aug; 95(6):e718-e732. PubMed ID: 32518148 [TBL] [Abstract][Full Text] [Related]
11. Pompe disease: early diagnosis and early treatment make a difference. Chien YH; Hwu WL; Lee NC Pediatr Neonatol; 2013 Aug; 54(4):219-27. PubMed ID: 23632029 [TBL] [Abstract][Full Text] [Related]
12. A new assay for fast, reliable CRIM status determination in infantile-onset Pompe disease. Wang Z; Okamoto P; Keutzer J Mol Genet Metab; 2014 Feb; 111(2):92-100. PubMed ID: 24044919 [TBL] [Abstract][Full Text] [Related]
13. A Race Against Time-Changing the Natural History of CRIM Negative Infantile Pompe Disease. Gupta P; Shayota BJ; Desai AK; Kiblawi F; Myridakis D; Messina J; Tah P; Tambini-King L; Kishnani PS Front Immunol; 2020; 11():1929. PubMed ID: 33013846 [TBL] [Abstract][Full Text] [Related]
14. Brain development in infantile-onset Pompe disease treated by enzyme replacement therapy. Chien YH; Lee NC; Peng SF; Hwu WL Pediatr Res; 2006 Sep; 60(3):349-52. PubMed ID: 16857770 [TBL] [Abstract][Full Text] [Related]
15. Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase. van Capelle CI; Poelman E; Frohn-Mulder IM; Koopman LP; van den Hout JMP; Régal L; Cools B; Helbing WA; van der Ploeg AT Int J Cardiol; 2018 Oct; 269():104-110. PubMed ID: 30049495 [TBL] [Abstract][Full Text] [Related]
16. Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy. Desai AK; Kazi ZB; Bali DS; Kishnani PS Mol Genet Metab Rep; 2019 Sep; 20():100475. PubMed ID: 31193175 [TBL] [Abstract][Full Text] [Related]
17. Classic infantile Pompe patients approaching adulthood: a cohort study on consequences for the brain. Ebbink BJ; Poelman E; Aarsen FK; Plug I; Régal L; Muentjes C; van der Beek NAME; Lequin MH; van der Ploeg AT; van den Hout JMP Dev Med Child Neurol; 2018 Jun; 60(6):579-586. PubMed ID: 29573408 [TBL] [Abstract][Full Text] [Related]
18. Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease. Rafael Bretón Martínez J; Martínez AC J Neurol Sci; 2015 Nov; 358(1-2):459-60. PubMed ID: 26279333 [No Abstract] [Full Text] [Related]
19. 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]
20. High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient. Rairikar M; Kazi ZB; Desai A; Walters C; Rosenberg A; Kishnani PS Mol Genet Metab; 2017 Sep; 122(1-2):76-79. PubMed ID: 28648664 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]