Identification of Ezetimibe and Pranlukast as Pharmacological Chaperones for the Treatment of the Rare Disease Mucopolysaccharidosis Type IVA
Author:
Affiliation:
1. Pharmacy Department, Faculty of Science, Universidad Nacional de Colombia, Bogotá D.C. 111321, Colombia
2. National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland 20892, United States
Funder
Departamento Administrativo de Ciencia, Tecnolog??a e Innovaci??n
U.S. Department of Health and Human Services
Fulbright Colombia
Pontificia Universidad Javeriana
Publisher
American Chemical Society (ACS)
Subject
Drug Discovery,Molecular Medicine
Link
https://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.9b00428
Reference86 articles.
1. A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases
2. Mucopolysaccharidosis IVA and glycosaminoglycans
3. Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations
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