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Last Updated: 10/31/2025
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Found 239 publications
Correction: Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry.
Journal: Journal of neurology
Published: July 31, 2025
Enzyme replacement therapies in adults with Pompe disease: from trials to real-world data.
Journal: Current opinion in neurology
Published: June 05, 2025
Marked Improvements in Airway Abnormalities and Multifaceted Outcomes After 2 Years Switching to Avalglucosidase Alfa: Evaluation of A 19-Year-Old Male Diagnosed With Late-Onset Pompe Disease.
Journal: American journal of medical genetics. Part A
Published: May 27, 2025
Defining clinically meaningful thresholds for forced vital capacity in patients with neuromuscular disorders: Lessons learned from the COMET study in Pompe disease.
Journal: Journal of neuromuscular diseases
Published: May 21, 2025
Improving the treatment of Pompe disease with enzyme replacement therapy: current strategies and clinical evidence.
Journal: Expert opinion on pharmacotherapy
Published: April 16, 2025
Enzyme replacement therapy for the treatment of late onset Pompe disease: A systematic review and network meta-analysis.
Journal: Orphanet journal of rare diseases
Published: March 29, 2025
Efficacy of avalglucosidase alfa in infantile-onset Pompe disease with high anti-alglucosidase alfa antibody titers.
Journal: Pediatrics international : official journal of the Japan Pediatric Society
Published: March 09, 2025
Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements.
Journal: European journal of hospital pharmacy : science and practice
Published: March 08, 2025
Analysis of the Italian cohort of late-onset Pompe disease (LOPD) patients after 10 and 15 years of therapy with alglucosidase alfa.
Journal: Journal of neurology
Published: March 03, 2025
Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL (S21.003).
Journal: Neurology
Published: February 20, 2025
Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial.
Journal: Journal of neurology
Published: February 05, 2025
Unexplained Progressive Respiratory Insufficiency and Weakness Diagnosed as Late-Onset Pompe Disease Through Biochemical and Molecular Genetic Testing.
Journal: The Neurohospitalist
Published: February 01, 2025
Last Updated: 10/31/2025