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Research article

First published 1997-09

Retroviral Transfer of Acid α -Glucosidase cDNA to Enzyme-Deficient Myoblasts Results in Phenotypic Spread of the Genotypic Correction by Both Secretion and Fusion

Joseph Z. Zaretsky, Fabio Candotti, Cornelius Boerkoel [...]

Human Gene Therapy

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Research article

First published 2001-03

Intercellular Transfer of the Virally Derived Precursor Form of Acid α-Glucosidase Corrects the Enzyme Deficiency in Inherited Cardioskeletal Myopathy Pompe Disease

Daniel F. Pauly, Thomas J. Fraites, Catalin Toma [...]

Human Gene Therapy

Free access

Letter

First published 2009-2

Donor-derived Hematopoietic Cell Contribution to Myofibers in Acid α-Glucosidase Deficiency

Zipora Yablonka-Reuveni

Journal of Histochemistry & Cytochemistry

Free access

Letter

First published 2009-2

Author's Response to “Donor-derived Hematopoietic Cell Contribution to Myofibers in Acid α-Glucosidase Deficiency: A Promising Progress or Back to the Beginning?”

Jun Mori

Journal of Histochemistry & Cytochemistry

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Research article

First published 2014-6

Immune Tolerance Induced Using Plasma Exchange and Rituximab in an Infantile Pompe Disease Patient

Federica Deodato, Virginia Maria Ginocchio, Alfredo Onofri [...]

Journal of Child Neurology

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Research article

First published 2008-06

Ability of Adeno-Associated Virus Serotype 8-Mediated Hepatic Expression of Acid α-Glucosidase to Correct the Biochemical and Motor Function Deficits of Presymptomatic and Symptomatic Pompe Mice

Robin J. Ziegler, Scott D. Bercury, Jonathan Fidler [...]

Human Gene Therapy

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Research article

First published 1992-08

Preparation of Monoclonal Antibodies Against Acid α-D-Glucosidase for Study of Chinese Glycogenosis Type II Patients

Ching-Yuang Lin, Shwu-Yea Lee, Zo-Nan Chang [...]

Monoclonal Antibodies in Immunodiagnosis & Immunotherapy

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Research article

First published 1997-01

Skeletal Muscle Weakness and Dysphagia Caused by Acid Maltase Deficiency: Nutritional Consequences of Coincident Celiac Sprue

Timothy S. King, Janice R. Anderson, E. Philip Wraight [...]

Journal of Parenteral and Enteral Nutrition

Open access

Brief report

First published 2024-03-05

Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa

Matthias Boentert, Emmanuelle Salort Campana, Shahram Attarian [...]

Journal of Neuromuscular Diseases

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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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