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Last Updated: 01/07/2026

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Found 2664 publications

Correction: Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Correction: Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
Published: December 15, 2025

A respiratory signature of disease progression in the Pompe rat.

A respiratory signature of disease progression in the Pompe rat.

Journal: Journal of neurophysiology
Published: December 05, 2025

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
Published: November 09, 2025

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
Published: November 09, 2025

A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.

A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.

Journal: Journal of neuromuscular diseases
Published: October 28, 2025

Dermatomyositis masking late onset Pompe disease in a patient with proximal muscle weakness.

Dermatomyositis masking late onset Pompe disease in a patient with proximal muscle weakness.

Journal: Journal of neuromuscular diseases
Published: October 22, 2025

Editorial Note: The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease.

Editorial Note: The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease.

Journal: PloS one
Published: October 22, 2025

Conference proceedings from the Western Canadian Neuromuscular Conference (WCNMC) - September 27-29, 2024, Calgary, Canada.

Conference proceedings from the Western Canadian Neuromuscular Conference (WCNMC) - September 27-29, 2024, Calgary, Canada.

Journal: Journal of neuromuscular diseases
Published: October 21, 2025

Neuromuscular diseases in pediatrics with specific treatments

Neuromuscular diseases in pediatrics with specific treatments

Journal: Medicina
Published: October 02, 2025

Anaesthetic Management of Advanced Late-Onset Pompe Disease: Challenges in a Major Abdominal Surgery.

Anaesthetic Management of Advanced Late-Onset Pompe Disease: Challenges in a Major Abdominal Surgery.

Journal: Cureus
Published: September 27, 2025

Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy.

Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy.

Journal: DNA and cell biology reports
Published: September 19, 2025

In Thickness and in Health: Delayed-Onset Pompe Disease Resembling Hypertrophic Cardiomyopathy.

In Thickness and in Health: Delayed-Onset Pompe Disease Resembling Hypertrophic Cardiomyopathy.

Journal: Cureus
Published: September 17, 2025
Showing 1-12 of 2,664

Last Updated: 01/07/2026