Literature DB >> 29719368

Pharmacotherapy of Gaucher Disease: Current and Future Options.

Lunawati L Bennett, Chris Fellner.   

Abstract

The clinical manifestations of Gaucher disease, a rare genetic lysosomal storage disorder, are debilitating, and the neuronopathic forms of the disease are fatal. The authors describe the current and investigational therapies for treatment.

Entities:  

Year:  2018        PMID： 29719368      PMCID： PMC5912244     

Source DB:  PubMed          Journal:  P T        ISSN： 1052-1372


  67 in total

Review 1.  Targeting glycosylation as a therapeutic approach.

Authors:  Raymond A Dwek; Terry D Butters; Frances M Platt; Nicole Zitzmann
Journal:  Nat Rev Drug Discov       Date:  2002-01       Impact factor: 84.694

2.  Benefits from unearthing "a biochemical Rosetta Stone".

Authors:  Roscoe O Brady
Journal:  J Biol Chem       Date:  2010-10-28       Impact factor: 5.157

3.  In memoriam: Norman S. Radin (1920-2013).

Authors:  James A Shayman
Journal:  J Lipid Res       Date:  2013-07       Impact factor: 5.922

4.  Histone deacetylase inhibitors increase glucocerebrosidase activity in Gaucher disease by modulation of molecular chaperones.

Authors:  Chunzhang Yang; Shervin Rahimpour; Jie Lu; Karel Pacak; Barbara Ikejiri; Roscoe O Brady; Zhengping Zhuang
Journal:  Proc Natl Acad Sci U S A       Date:  2012-12-31       Impact factor: 11.205

Review 5.  Gaucher disease and other storage disorders.

Authors:  Gregory A Grabowski
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2012

6.  Cognitive outcome in treated patients with chronic neuronopathic Gaucher disease.

Authors:  Ozlem Goker-Alpan; Edythe A Wiggs; Michael J Eblan; William Benko; Shira G Ziegler; Ellen Sidransky; Raphael Schiffmann
Journal:  J Pediatr       Date:  2008-02-14       Impact factor: 4.406

Review 7.  The design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?

Authors:  James A Shayman
Journal:  Trans Am Clin Climatol Assoc       Date:  2013

Review 8.  Gaucher disease: mutation and polymorphism spectrum in the glucocerebrosidase gene (GBA).

Authors:  Kathleen S Hruska; Mary E LaMarca; C Ronald Scott; Ellen Sidransky
Journal:  Hum Mutat       Date:  2008-05       Impact factor: 4.878

9.  Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease.

Authors:  M Biegstraaten; T M Cox; N Belmatoug; M G Berger; T Collin-Histed; S Vom Dahl; M Di Rocco; C Fraga; F Giona; P Giraldo; M Hasanhodzic; D A Hughes; P O Iversen; A I Kiewiet; E Lukina; M Machaczka; T Marinakis; E Mengel; G M Pastores; U Plöckinger; H Rosenbaum; C Serratrice; A Symeonidis; J Szer; J Timmerman; A Tylki-Szymańska; M Weisz Hubshman; D I Zafeiriou; A Zimran; C E M Hollak
Journal:  Blood Cells Mol Dis       Date:  2016-10-24       Impact factor: 3.039

10.  Glycosylation and functionality of recombinant β-glucocerebrosidase from various production systems.

Authors:  Yoram Tekoah; Salit Tzaban; Tali Kizhner; Mariana Hainrichson; Anna Gantman; Myriam Golembo; David Aviezer; Yoseph Shaaltiel
Journal:  Biosci Rep       Date:  2013-09-25       Impact factor: 3.840
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  2 in total

Review 1.  Gaucher disease - more than just a rare lipid storage disease.

Authors:  Jaehyeok Roh; Subbaya Subramanian; Neal J Weinreb; Reena V Kartha
Journal:  J Mol Med (Berl)       Date:  2022-01-23       Impact factor: 4.599

2.  Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).

Authors:  Derralynn A Hughes; Patrick Deegan; Pilar Giraldo; Özlem Göker-Alpan; Heather Lau; Elena Lukina; Shoshana Revel-Vilk; Maurizio Scarpa; Jaco Botha; Noga Gadir; Ari Zimran
Journal:  J Clin Med       Date:  2022-08-31       Impact factor: 4.964
  2 in total

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