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First published 2009-05

Progress in enzyme replacement therapy in glycogen storage disease type II

Corrado Angelini, Claudio Semplicini, Paola Tonin [...]

Therapeutic Advances in Neurological Disorders

Free access

Research article

First published 2018

Low Prevalence Estimates of Late-Onset Glycogen Storage Disease Type II in French-Speaking Belgium are not Due to Missed Diagnoses

Gauthier Remiche, Zoltan Lukacs, David C. Kasper [...]

Journal of Neuromuscular Diseases

Free access

Case report

First published 2007-01

Evidence of cardiomyocyte necrosis in glycogen storage disease type II

Annals of Clinical Biochemistry: International Journal of Laboratory Medicine

Retricted access

Research article

First published 2001-05

Long-Term Efficacy after [E1 -,polymerase - ] Adenovirus-Mediated Transfer of Human Acid-α-Glucosidase Gene into Glycogen Storage Disease Type II Knockout Mice

E.Y. Ding, B.L. Hodges, H. Hu [...]

Human Gene Therapy

Retricted access

Research article

First published 2005-01

Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid α-Glucosidase in a Model of Glycogen Storage Disease Type II

Kerry O. Cresawn, Thomas J. Fraites, Clive Wasserfall [...]

Human Gene Therapy

Open access

Research article

First published 2015-3

First Case of Late-Onset Glycogen Storage Disease Type II in Russia with a Novel Mutation

S.S. Nikitin, M.O. Kovalchuk, E.A. Proskurina [...]

Journal of Neuromuscular Diseases

Retricted access

Other

First published 2010-8

Screening of Late-Onset Pompe Disease in a Sample of Mexican Patients With Myopathies of Unknown Etiology: Identification of a Novel Mutation in the Acid α-Glucosidase Gene

Miguel Angel Alcántara-Ortigoza, Ariadna González-del Angel, Rehotbevely Barrientos-Ríos [...]

Journal of Child Neurology

Free access

Research article

First published 2014-8

Effects of respiratory muscle training (RMT) in children with infantile-onset Pompe disease and respiratory muscle weakness

Harrison N. Jones, Kelly D. Crisp, Tronda Moss [...]

Journal of Pediatric Rehabilitation Medicine

Free access

Case report

First published 2020-4-13

Training,detraining,and retraining: Two 12-week respiratory muscle training regimens in a child with infantile-onset Pompe disease

Kelly D. Crisp, Laura E. Case, Richard M. Kravitz [...]

Journal of Pediatric Rehabilitation Medicine

Retricted access

Research article

First published 2012-3

Infantile Pompe Disease: Clinical and Genetic Characteristics With an Experience of Enzyme Replacement Therapy

Anna Cho, Su Jin Kim, Byung Chan Lim [...]

Journal of Child Neurology

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