A Chaperone Enhances Blood α-Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy-Reference-Cited by-同舟云学术

A Chaperone Enhances Blood α-Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy

Published:2014-11 Issue:11 Volume:22 Page:2004-2012
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Parenti Giancarlo,Fecarotta Simona,la Marca Giancarlo,Rossi Barbara,Ascione Serena,Donati Maria Alice,Morandi Lucia Ovidia,Ravaglia Sabrina,Pichiecchio Anna,Ombrone Daniela,Sacchini Michele,Pasanisi Maria Barbara,De Filippi Paola,Danesino Cesare,Della Casa Roberto,Romano Alfonso,Mollica Carmine,Rosa Margherita,Agovino Teresa,Nusco Edoardo,Porto Caterina,Andria Generoso

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference41 articles.

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2. Autophagy in skeletal muscle: implications for Pompe disease;Shea;Int J Clin Pharmacol Ther,2009

3. Neural deficits contribute to respiratory insufficiency in Pompe disease;DeRuisseau;Proc Natl Acad Sci USA,2009

4. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease;Falk;Mol Ther,2013

5. Recombinant human alpha-glucosidase from rabbit milk in Pompe patients;Van den Hout;Lancet,2000

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