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Journal Abstract Search

296 related items for PubMed ID: 24380126

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

  • 2. 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]

  • 3. 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]

  • 4. [Enzyme replacement therapy of lysosomal storage diseases].
    Germain DP, Boucly C, Carlier RY, Caudron E, Charlier P, Colas F, Jabbour F, Martinez V, Mokhtari S, Orlikowski D, Pellegrini N, Perronne C, Prigent H, Rubinsztajn R, Benistan K.
    Rev Med Interne; 2010 Dec 01; 31 Suppl 2():S279-91. PubMed ID: 21211680
    [Abstract] [Full Text] [Related]

  • 5. Enzyme enhancement therapeutics for lysosomal storage diseases: Current status and perspective.
    Thomas R, Kermode AR.
    Mol Genet Metab; 2019 Feb 01; 126(2):83-97. PubMed ID: 30528228
    [Abstract] [Full Text] [Related]

  • 6. Enzyme replacement therapy for lysosomal storage diseases.
    Lachmann RH.
    Curr Opin Pediatr; 2011 Dec 01; 23(6):588-93. PubMed ID: 21946346
    [Abstract] [Full Text] [Related]

  • 7. New strategies for the treatment of lysosomal storage diseases (review).
    Parenti G, Pignata C, Vajro P, Salerno M.
    Int J Mol Med; 2013 Jan 01; 31(1):11-20. PubMed ID: 23165354
    [Abstract] [Full Text] [Related]

  • 8. Therapeutic approaches for lysosomal storage diseases: a patent update.
    Urbanelli L, Sagini K, Polidoro M, Brozzi A, Magini A, Emiliani C.
    Recent Pat CNS Drug Discov; 2013 Aug 01; 8(2):91-109. PubMed ID: 23713988
    [Abstract] [Full Text] [Related]

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  • 10. Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges.
    Desnick RJ, Schuchman EH.
    Annu Rev Genomics Hum Genet; 2012 Aug 01; 13():307-35. PubMed ID: 22970722
    [Abstract] [Full Text] [Related]

  • 11. Treatments for lysosomal storage disorders.
    Lachmann R.
    Biochem Soc Trans; 2010 Dec 01; 38(6):1465-8. PubMed ID: 21118108
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  • 14. Enzyme replacement therapy and substrate reduction therapy in lysosomal storage disorders with neurological expression.
    Valayannopoulos V.
    Handb Clin Neurol; 2013 Dec 01; 113():1851-7. PubMed ID: 23622408
    [Abstract] [Full Text] [Related]

  • 15. 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]

  • 16. Orphan drug development.
    Pastores GM, Gupta P.
    Pediatr Endocrinol Rev; 2013 Nov 01; 11 Suppl 1():64-7. PubMed ID: 24380124
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  • 18. Recent developments in therapeutic approaches for lysosomal storage diseases.
    Urbanelli L, Magini A, Polchi A, Polidoro M, Emiliani C.
    Recent Pat CNS Drug Discov; 2011 Jan 01; 6(1):1-19. PubMed ID: 21073432
    [Abstract] [Full Text] [Related]

  • 19. Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification.
    Keyzor I, Shohet S, Castelli J, Sitaraman S, Veleva-Rotse B, Weimer JM, Fox B, Willer T, Tuske S, Crathorne L, Belzar KJ.
    Biomolecules; 2023 Aug 07; 13(8):. PubMed ID: 37627292
    [Abstract] [Full Text] [Related]

  • 20. Gene therapy for the lysosomal storage disorders.
    Cabrera-Salazar MA, Novelli E, Barranger JA.
    Curr Opin Mol Ther; 2002 Aug 07; 4(4):349-58. PubMed ID: 12222873
    [Abstract] [Full Text] [Related]

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