Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy-Reference-Cited by-同舟云学术

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Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy

Published:2010-12 Issue:4 Volume:101 Page:324-331
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Raben Nina,Ralston Evelyn,Chien Yin-Hsiu,Baum Rebecca,Schreiner Cynthia,Hwu Wuh-Liang,Zaal Kristien J.M.,Plotz Paul H.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference38 articles.

1. Autophagy revisited: a conversation with Christian de Duve;Klionsky;Autophagy,2008

2. Glycogen storage disease type II: acid alpha-glucosidase (acid maltase) deficiency;Hirschhorn,2001

3. Acid Maltase Deficiency;Engel,2003

4. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease;Kishnani;J. Pediatr.,2006

5. The natural course of non-classic Pompe's disease; a review of 225 published cases;Winkel;J. Neurol.,2005

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1. Newborn Screening for Neuromuscular Disorders, Disorders of Glycogen Metabolism, and Fatty Acid Oxidation;OBM Genetics;2024-04-18

2. Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease;2024-02-22

3. Evaluating avalglucosidase alfa for the management of late-onset Pompe disease;Expert Review of Neurotherapeutics;2024-01-23

4. An analysis of Pompe newborn screening data: a new prevalence at birth, insight and discussion;Frontiers in Pediatrics;2024-01-08

5. Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice;2023-12-28

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