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 *

161 related articles for article (PubMed ID: 24380122)

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

  • 2. Ten plus one challenges in diseases of the lysosomal system.
    Grabowski GA; Whitley C
    Mol Genet Metab; 2017; 120(1-2):38-46. PubMed ID: 27923545
    [TBL] [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
    [TBL] [Abstract][Full Text] [Related]  

  • 4. [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; 18(10):1119-23. PubMed ID: 21873040
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Treating lysosomal storage disorders: What have we learnt?
    Lachmann RH
    J Inherit Metab Dis; 2020 Jan; 43(1):125-132. PubMed ID: 31140601
    [TBL] [Abstract][Full Text] [Related]  

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

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

  • 8. The role of antibodies in enzyme treatments and therapeutic strategies.
    Bigger BW; Saif M; Linthorst GE
    Best Pract Res Clin Endocrinol Metab; 2015 Mar; 29(2):183-94. PubMed ID: 25987172
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Enzyme Replacement Therapy for Lysosomal Storage Diseases.
    Kleppin S
    J Infus Nurs; 2020; 43(5):243-245. PubMed ID: 32881810
    [No Abstract]   [Full Text] [Related]  

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

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

  • 12. Overcoming the Next Barriers to Successful Therapy.
    Cohen IJ; Baris H; Mistry PK; Sands MS
    Pediatr Endocrinol Rev; 2016 Jun; 13 Suppl 1():629. PubMed ID: 27491209
    [No Abstract]   [Full Text] [Related]  

  • 13. Orphan drug development.
    Pastores GM; Gupta P
    Pediatr Endocrinol Rev; 2013 Nov; 11 Suppl 1():64-7. PubMed ID: 24380124
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Roscoe Owen Brady, MD: Remembrances of co-investigators and colleagues.
    Desnick RJ; Barton NW; Furbish S; Grabowski GA; Karlsson S; Kolodny EH; Medin JA; Murray GJ; Mistry PK; Patterson MC; Schiffmann R; Weinreb NJ
    Mol Genet Metab; 2017; 120(1-2):1-7. PubMed ID: 27866832
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Emptying the stores: lysosomal diseases and therapeutic strategies.
    Platt FM
    Nat Rev Drug Discov; 2018 Feb; 17(2):133-150. PubMed ID: 29147032
    [TBL] [Abstract][Full Text] [Related]  

  • 16. The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease.
    Hollak CE; Weinreb NJ
    Best Pract Res Clin Endocrinol Metab; 2015 Mar; 29(2):205-18. PubMed ID: 25987174
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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; 13():307-35. PubMed ID: 22970722
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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; 13(8):. PubMed ID: 37627292
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Pulmonary involvement in selected lysosomal storage diseases and the impact of enzyme replacement therapy: A state-of-the art review.
    Jezela-Stanek A; Chorostowska-Wynimko J; Tylki-Szymańska A
    Clin Respir J; 2020 May; 14(5):422-429. PubMed ID: 31912638
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Treatment for Lsds: real options for several diseases. Forward.
    Cohen IJ; Baris HN; Mistry PK; Sands MS
    Pediatr Endocrinol Rev; 2014 Sep; 12 Suppl 1():71. PubMed ID: 25345087
    [No Abstract]   [Full Text] [Related]  

    [Next]    [New Search]
    of 9.