BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

614 related articles for article (PubMed ID: 23787031)

  • 1. Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.
    Prater SN; Patel TT; Buckley AF; Mandel H; Vlodavski E; Banugaria SG; Feeney EJ; Raben N; Kishnani PS
    Orphanet J Rare Dis; 2013 Jun; 8():90. PubMed ID: 23787031
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.
    Yi H; Sun T; Armstrong D; Borneman S; Yang C; Austin S; Kishnani PS; Sun B
    J Mol Med (Berl); 2017 May; 95(5):513-521. PubMed ID: 28154884
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.
    Van den Hout JM; Kamphoven JH; Winkel LP; Arts WF; De Klerk JB; Loonen MC; Vulto AG; Cromme-Dijkhuis A; Weisglas-Kuperus N; Hop W; Van Hirtum H; Van Diggelen OP; Boer M; Kroos MA; Van Doorn PA; Van der Voort E; Sibbles B; Van Corven EJ; Brakenhoff JP; Van Hove J; Smeitink JA; de Jong G; Reuser AJ; Van der Ploeg AT
    Pediatrics; 2004 May; 113(5):e448-57. PubMed ID: 15121988
    [TBL] [Abstract][Full Text] [Related]  

  • 5. CRIM-negative infantile Pompe disease: 42-month treatment outcome.
    Rohrbach M; Klein A; Köhli-Wiesner A; Veraguth D; Scheer I; Balmer C; Lauener R; Baumgartner MR
    J Inherit Metab Dis; 2010 Dec; 33(6):751-7. PubMed ID: 20882352
    [TBL] [Abstract][Full Text] [Related]  

  • 6. The value of muscle biopsies in Pompe disease: identifying lipofuscin inclusions in juvenile- and adult-onset patients.
    Feeney EJ; Austin S; Chien YH; Mandel H; Schoser B; Prater S; Hwu WL; Ralston E; Kishnani PS; Raben N
    Acta Neuropathol Commun; 2014 Jan; 2():2. PubMed ID: 24383498
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease.
    Cousens LP; Mingozzi F; van der Marel S; Su Y; Garman R; Ferreira V; Martin W; Scott DW; De Groot AS
    Hum Vaccin Immunother; 2012 Oct; 8(10):1459-64. PubMed ID: 23095864
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Benefits of Prophylactic Short-Course Immune Tolerance Induction in Patients With Infantile Pompe Disease: Demonstration of Long-Term Safety and Efficacy in an Expanded Cohort.
    Desai AK; Baloh CH; Sleasman JW; Rosenberg AS; Kishnani PS
    Front Immunol; 2020; 11():1727. PubMed ID: 32849613
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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]  

  • 13. CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy.
    Berrier KL; Kazi ZB; Prater SN; Bali DS; Goldstein J; Stefanescu MC; Rehder CW; Botha EG; Ellaway C; Bhattacharya K; Tylki-Szymanska A; Karabul N; Rosenberg AS; Kishnani PS
    Genet Med; 2015 Nov; 17(11):912-8. PubMed ID: 25741864
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. 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]  

  • 16. 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]  

  • 17. 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]  

  • 18. Long-term follow-up results in enzyme replacement therapy for Pompe disease: a case report.
    Del Rizzo M; Fanin M; Cerutti A; Cazzorla C; Milanesi O; Nascimbeni AC; Angelini C; Giordano L; Bordugo A; Burlina AB
    J Inherit Metab Dis; 2010 Dec; 33 Suppl 3():S389-93. PubMed ID: 20830524
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Autophagy in skeletal muscle: implications for Pompe disease.
    Shea L; Raben N
    Int J Clin Pharmacol Ther; 2009; 47 Suppl 1(Suppl 1):S42-7. PubMed ID: 20040311
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 31.