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Journal Abstract Search

346 related items for PubMed ID: 21946346

  • 1. Enzyme replacement therapy for lysosomal storage diseases.
    Lachmann RH.
    Curr Opin Pediatr; 2011 Dec; 23(6):588-93. PubMed ID: 21946346
    [Abstract] [Full Text] [Related]

  • 2. Novel treatments and future perspectives: outcomes of intrathecal drug delivery.
    Dickson PI.
    Int J Clin Pharmacol Ther; 2009 Dec; 47 Suppl 1():S124-7. PubMed ID: 20040323
    [Abstract] [Full Text] [Related]

  • 3. Enzyme replacement therapy for lysosomal storage diseases.
    Ohashi T.
    Pediatr Endocrinol Rev; 2012 Oct; 10 Suppl 1():26-34. PubMed ID: 23330243
    [Abstract] [Full Text] [Related]

  • 4. Targeting macrophages with baculovirus-produced lysosomal enzymes: implications for enzyme replacement therapy of the glycoprotein storage disorder galactosialidosis.
    Bonten EJ, Wang D, Toy JN, Mann L, Mignardot A, Yogalingam G, D'Azzo A.
    FASEB J; 2004 Jun; 18(9):971-3. PubMed ID: 15084520
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  • 7. Reversibility of cellular and organ pathology in enzyme replacement trials in animal models of lysosomal storage diseases.
    Gieselmann V.
    Acta Paediatr Suppl; 2006 Apr; 95(451):93-9. PubMed ID: 16720473
    [Abstract] [Full Text] [Related]

  • 8. Reversal of peripheral and central neural storage and ataxia after recombinant enzyme replacement therapy in alpha-mannosidosis mice.
    Blanz J, Stroobants S, Lüllmann-Rauch R, Morelle W, Lüdemann M, D'Hooge R, Reuterwall H, Michalski JC, Fogh J, Andersson C, Saftig P.
    Hum Mol Genet; 2008 Nov 15; 17(22):3437-45. PubMed ID: 18713755
    [Abstract] [Full Text] [Related]

  • 9. Oral small molecule therapy for lysosomal storage diseases.
    Weinreb NJ.
    Pediatr Endocrinol Rev; 2013 Nov 15; 11 Suppl 1():77-90. PubMed ID: 24380126
    [Abstract] [Full Text] [Related]

  • 10. Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy.
    Stroobants S, Gerlach D, Matthes F, Hartmann D, Fogh J, Gieselmann V, D'Hooge R, Matzner U.
    Hum Mol Genet; 2011 Jul 15; 20(14):2760-9. PubMed ID: 21515587
    [Abstract] [Full Text] [Related]

  • 11. Recent progress in development of transgenic silkworms overexpressing recombinant human proteins with therapeutic potential in silk glands.
    Itoh K, Kobayashi I, Nishioka S, Sezutsu H, Machii H, Tamura T.
    Drug Discov Ther; 2016 Feb 15; 10(1):34-9. PubMed ID: 26971553
    [Abstract] [Full Text] [Related]

  • 12. Treatment of lysosomal storage diseases: recent patents and future strategies.
    Ortolano S, Viéitez I, Navarro C, Spuch C.
    Recent Pat Endocr Metab Immune Drug Discov; 2014 Jan 15; 8(1):9-25. PubMed ID: 24433521
    [Abstract] [Full Text] [Related]

  • 13. [Enzyme replacement therapy for lysosomal storage disorders].
    Valayannopoulos V, Brassier A, Chabli A, Caillaud C, Lemoine M, Odent T, Arnoux JB, de Lonlay P.
    Arch Pediatr; 2011 Oct 15; 18(10):1119-23. PubMed ID: 21873040
    [Abstract] [Full Text] [Related]

  • 14. Short-term, high dose enzyme replacement therapy in sialidosis mice.
    Wang D, Bonten EJ, Yogalingam G, Mann L, d'Azzo A.
    Mol Genet Metab; 2005 Jul 15; 85(3):181-9. PubMed ID: 15979029
    [Abstract] [Full Text] [Related]

  • 15. Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice.
    Shen JS, Busch A, Day TS, Meng XL, Yu CI, Dabrowska-Schlepp P, Fode B, Niederkrüger H, Forni S, Chen S, Schiffmann R, Frischmuth T, Schaaf A.
    J Inherit Metab Dis; 2016 Mar 15; 39(2):293-303. PubMed ID: 26310963
    [Abstract] [Full Text] [Related]

  • 16. Enzyme replacement therapy for lysosomal storage disorders: successful transition from concept to clinical practice.
    Sly WS.
    Mo Med; 2004 Mar 15; 101(2):100-4. PubMed ID: 15119106
    [Abstract] [Full Text] [Related]

  • 17. Enzyme Replacement Therapy: A Review and Its Role in Treating Lysosomal Storage Diseases.
    Li M.
    Pediatr Ann; 2018 May 01; 47(5):e191-e197. PubMed ID: 29750286
    [Abstract] [Full Text] [Related]

  • 18. Glyco-engineering strategies for the development of therapeutic enzymes with improved efficacy for the treatment of lysosomal storage diseases.
    Oh DB.
    BMB Rep; 2015 Aug 01; 48(8):438-44. PubMed ID: 25999178
    [Abstract] [Full Text] [Related]

  • 19. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I.
    Kakkis E, McEntee M, Vogler C, Le S, Levy B, Belichenko P, Mobley W, Dickson P, Hanson S, Passage M.
    Mol Genet Metab; 2004 Aug 01; 83(1-2):163-74. PubMed ID: 15464431
    [Abstract] [Full Text] [Related]

  • 20. Treatment for LSDs: no longer just enzyme replacement therapy for Gaucher disease. Foreword.
    Cohen IJ, Baris H, Mistry PK.
    Pediatr Endocrinol Rev; 2013 Nov 01; 11 Suppl 1():58. PubMed ID: 24380122
    [No Abstract] [Full Text] [Related]

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