Therapeutic Strategies for Gaucher Disease: Miglustat (NB-DNJ) as a Pharmacological Chaperone for Glucocerebrosidase and the Different Thermostability of Velaglucerase Alfa and Imiglucerase-Reference-Cited by-同舟云学术

Therapeutic Strategies for Gaucher Disease: Miglustat (NB-DNJ) as a Pharmacological Chaperone for Glucocerebrosidase and the Different Thermostability of Velaglucerase Alfa and Imiglucerase

Published:2011-11-03 Issue:6 Volume:8 Page:2390-2397
ISSN:1543-8384
Container-title:Molecular Pharmaceutics
language:en
Short-container-title:Mol. Pharmaceutics

Author:

Abian Olga¹²³,Alfonso Pilar¹²,Velazquez-Campoy Adrian³⁴⁵,Giraldo Pilar¹²⁶,Pocovi Miguel²⁴,Sancho Javier³⁴

Affiliation:

1. Unidad de Investigación Traslacional, Miguel Servet Universitary Hospital, Zaragoza, Spain

2. Aragon Health Sciences Institute (I+CS), Zaragoza, Spain

3. Institute of Biocomputation and Physics of Complex Systems (BIFI), University of Zaragoza, BIFI-IQFR (CSIC)-Joint Unit, Spain

4. Department of Biochemistry and Cellular and Molecular Biology, University of Zaragoza, Spain

5. Fundación ARAID (ARAID-BIFI), Diputación General de Aragón, Spain

6. Service of Hematology, Miguel Servet Universitary Hospital, Zaragoza, Spain

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Pharmaceutical Science,Molecular Medicine

Link

https://pubs.acs.org/doi/pdf/10.1021/mp200313e

Reference65 articles.

1. Metabolism of glucocerebrosides II. Evidence of an enzymatic deficiency in Gaucher's disease

2. New directions in the treatment of Gaucher disease

3. Therapeutic strategies to ameliorate lysosomal storage disorders – a focus on Gaucher disease

4. Therapy for the Sphingolipidoses

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