4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations-Reference-Cited by-同舟云学术

4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations

Published:2018-11 Issue:20 Volume:26 Page:5462-5469
ISSN:0968-0896
Container-title:Bioorganic & Medicinal Chemistry
language:en
Short-container-title:Bioorganic & Medicinal Chemistry

Author:

Front Sophie,Almeida Sofia,Zoete Vincent,Charollais-Thoenig Julie,Gallienne Estelle,Marmy Céline,Pilloud Vincent^ORCID,Marti Roger^ORCID,Wood Tim,Martin Olivier R.,Demotz Stéphane

Funder

Swiss Sanfilippo Foundation

Publisher

Elsevier BV

Subject

Organic Chemistry,Clinical Biochemistry,Drug Discovery,Pharmaceutical Science,Molecular Biology,Molecular Medicine,Biochemistry

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4. Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies;Germain;Orphanet J Rare Dis,2012

5. Chemical chaperone therapy for brain pathology in GM1-gangliosidosis;Matsuda;Proc Natl Acad Sci USA,2003

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