Sage Journals: Discover world-class research

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

Open access

Research article

First published 2022

Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort

Tina Duong, Priya S. Kishnani, Kristina An Haack [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2015-3

A Phase 4 Prospective Study in Patients with Adult Pompe Disease Treated with Alglucosidase Alfa

Beth L. Thurberg, Pierre Carlier, John T. Kissel [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2015-3

A 4-Year Observational Clinical Study of 24 Adult Pompe Disease Patients under Alglucosidase Alfa Enzyme Replacement Therapy

Karolina M. Stepien, Jane Whitby, Mark Roberts [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2026

Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease

Robert J. Hopkin, Barry J. Byrne, Mazen M. Dimachkie [...]

Therapeutic Advances in Rare Disease

Open access

Review article

First published 2017-5-25

Lysosomal storage diseases

Carlos R. Ferreira, William A. Gahl

Translational Science of Rare Diseases

Retricted access

Research article

First published 2006-7

Anidulafungin

Dennis J. Cada, Terri Levien, Danial E. Baker

Hospital Pharmacy

Open access

Research article

First published 2025-7

Defining clinically meaningful thresholds for forced vital capacity in patients with neuromuscular disorders: Lessons learned from the COMET study in Pompe disease

Kenneth I Berger, Cristina Ivanescu, Jérôme Msihid [...]

Journal of Neuromuscular Diseases

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

Open access

Research article

First published 2024-01-02

Long-Term Outcome of Infantile Onset Pompe Disease Patients Treated with Enzyme Replacement Therapy - Data from a German-Austrian Cohort

Charlotte Pfrimmer, Martin Smitka, Nicole Muschol [...]

Journal of Neuromuscular Diseases

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