Magali Periquet

Chilly, France

Last Updated: 09/26/2026

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Found 13 publications

The Patient Experience of Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease Post-Diagnosis: Results from an International Survey.

The Patient Experience of Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease Post-Diagnosis: Results from an International Survey.

Journal: Neurology and therapy

Published: April 07, 2026

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.

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

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

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

Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial.

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

Corrigendum to "Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials" [Molecular Genetics and Metabolism, 143 (2024) 108608].

Corrigendum to "Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials" [Molecular Genetics and Metabolism, 143 (2024) 108608].

Journal: Molecular genetics and metabolism

Published: January 09, 2025

Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials.

Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials.

Journal: Molecular genetics and metabolism

Published: June 13, 2024

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.

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: February 22, 2024

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

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

Journal: Journal of neuromuscular diseases

Published: December 31, 2023

Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.

Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.

Journal: Molecular genetics and metabolism reports

Published: December 06, 2023

Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.

Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.

Journal: Orphanet journal of rare diseases

Published: April 20, 2023

Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study.

Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study.

Journal: Clinical pharmacology in drug development

Published: April 12, 2023

The Latin American experience with a next generation sequencing genetic panel for recessive limb-girdle muscular weakness and Pompe disease.

The Latin American experience with a next generation sequencing genetic panel for recessive limb-girdle muscular weakness and Pompe disease.

Journal: Orphanet journal of rare diseases

Published: September 10, 2019

Showing 1-12 of 13

Last Updated: 09/26/2026