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TARD , Celine

Last name: TARD

First name: Celine

April 9, 2024 (v1)

Journal article

Metadata-only

Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study

Tard, Celine Bouhour, F. Michaud, M.

IntroductionLate-onset Pompe disease (LOPD) is characterized by a progressive myopathy resulting from a deficiency of acid α-glucosidase enzyme activity. Enzyme replacement therapy has been shown to be effective, but long-term treatment results vary. Avalglucosidase alfa demonstrated non-inferiority to alglucosidase alfa in a phase 3 study,...

Uploaded on: September 7, 2024
2024 (v1)

Journal article

Metadata-only

Bulbar muscle impairment in patients with late onset Pompe disease: Insight from the French Pompe registry

Retailleau, Emilie Lefeuvre, Claire de Antonio, Marie

© 2024 The Author(s). European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.Background and purpose: Late onset Pompe disease (LOPD) is a rare neuromuscular disorder caused by a deficit in acid alpha-glucosidase. Macroglossia and swallowing disorders have already been reported, but no study...

Uploaded on: September 5, 2024