N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease-Reference-Cited by-同舟云学术

N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease

Published:2017-11-22 Issue:23 Volume:60 Page:9462-9469
ISSN:0022-2623
Container-title:Journal of Medicinal Chemistry
language:en
Short-container-title:J. Med. Chem.

Author:

D’Alonzo Daniele¹^ORCID,De Fenza Maria¹,Porto Caterina²,Iacono Roberta³,Huebecker Mylene⁴,Cobucci-Ponzano Beatrice³,Priestman David A.⁴,Platt Frances⁴,Parenti Giancarlo²⁵,Moracci Marco³⁶^ORCID,Palumbo Giovanni¹,Guaragna Annalisa¹^ORCID

Affiliation:

1. Department of Chemical Sciences, Università degli Studi di Napoli Federico II, via Cintia, 80126 Napoli, Italy

2. Department of Translational Medical Sciences, Section of Pediatrics, Università degli Studi di Napoli Federico II, Via S. Pansini 5, 80131 Napoli, Italy

3. Institute of Biosciences and Bioresources, Consiglio Nazionale delle Ricerche, Via P. Castellino 111, 80131 Napoli, Italy

4. Department of Pharmacology, University of Oxford, Mansfield Road, Oxford OX1 3QT, U.K.

5. Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, 80078 Pozzuoli, Italy

6. Department of Biology, Università degli Studi di Napoli Federico II, via Cintia, 80126 Napoli, Italy

Funder

Ministero dell’Istruzione, dell’Università e della Ricerca

Mizutani Foundation for Glycoscience

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Molecular Medicine

Link

https://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.7b00646

Reference40 articles.

1. Function and Structure Studies of GH Family 31 and 97 α-Glycosidases

2. Pompe's disease

3. Sphingolipid lysosomal storage disorders

4. Lysosomal Storage Diseases: From Pathophysiology to Therapy

5. Recombinant human α-glucosidase from rabbit milk in Pompe patients

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