Quantitative assessment of the exposure–efficacy relationship of glucocerebrosidase using Markovian elements in Gaucher patients treated with enzyme replacement therapy-Reference-Cited by-同舟云学术

Quantitative assessment of the exposure–efficacy relationship of glucocerebrosidase using Markovian elements in Gaucher patients treated with enzyme replacement therapy

Published:2022-01-10 Issue: Volume: Page:
ISSN:0306-5251
Container-title:British Journal of Clinical Pharmacology
language:en
Short-container-title:Brit J Clinical Pharma

Author:

Gras‐Colomer Elena¹,Mangas‐Sanjuán Víctor²³^ORCID,Martínez‐Gómez María‐Amparo⁴⁵,Climente‐Martí Mónica²⁴,Merino‐Sanjuan Matilde²³

Affiliation:

1. Pharmacy Department Hospital Manises of Valencia Valencia Spain

2. Department of Pharmacy Technology and Parasitology, Faculty of Pharmacy University of Valencia Valencia Spain

3. Interuniversity Institute of Recognition Research Molecular and Technological Development University of Valencia Valencia Spain

4. Pharmacy Department University Hospital Doctor Peset of Valencia Valencia Spain

5. Foundation for the Promotion of Healthcare and Biomedical Research in the Valencian Community (FISABIO) Valencia Spain

Funder

Generalitat Valenciana

Publisher

Wiley

Subject

Pharmacology (medical),Pharmacology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bcp.15198

Reference64 articles.

1. Enzymology, genetics, and treatment;Grabowski GA;Adv Hum Genet,1993

2. Therapeutic goals in the treatment of Gaucher disease;Pastores GM;Semin Hematol,2004

3. Gaucher disease;Beutler E;Curr Opin Hematol,1997

4. Glucocerebrosidase 2 gene deletion rescues type 1 Gaucher disease;Mistry PK;Proc Natl Acad Sci U S A,2014

5. Glucosylceramide transfer from lysosomes—the missing link in molecular pathology of glucosylceramidase deficiency: a hypothesis based on existing data;Elleder M;J Inherit Metab Dis,2006