These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

278 related articles for article (PubMed ID: 29050825)

  • 1. Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy.
    McIntosh PT; Hobson-Webb LD; Kazi ZB; Prater SN; Banugaria SG; Austin S; Wang R; Enterline DS; Frush DP; Kishnani PS
    Mol Genet Metab; 2018 Feb; 123(2):85-91. PubMed ID: 29050825
    [TBL] [Abstract][Full Text] [Related]  

  • 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]  

  • 4. 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]  

  • 5. 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]  

  • 6. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy.
    Chen HA; Hsu RH; Fang CY; Desai AK; Lee NC; Hwu WL; Tsai FJ; Kishnani PS; Chien YH
    Front Immunol; 2024; 15():1336599. PubMed ID: 38715621
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 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. 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]  

  • 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]
    of 14.