1. Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic.
- Author
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Tard C, Salort-Campana E, Michaud M, Spinazzi M, Nadaj Pakleza A, Durr H, Bouhour F, Lefeuvre C, Thomas R, Arrassi A, Taouagh N, Solé G, and Laforêt P
- Subjects
- Enzyme Replacement Therapy adverse effects, Humans, Pandemics, SARS-CoV-2, Treatment Outcome, alpha-Glucosidases therapeutic use, COVID-19, Glycogen Storage Disease Type II drug therapy
- Abstract
Background and Purpose: Data on interruption of enzyme replacement therapy (ERT) are scarce in late onset Pompe disease. Due to the COVID-19 crisis, eight neuromuscular reference centers in France were obligated to stop the treatment for 31 patients., Methods: We collected the motor and respiratory data from our French registry, before COVID-19 and at treatment restart., Results: In 2.2 months (mean), patients showed a significant deterioration of 37 m (mean) in the 6-min walk test and a loss of 210 ml (mean) of forced vital capacity, without ad integrum restoration after 3 months of ERT restart., Conclusions: This national study based on data from the French Pompe Registry shows that the interruption of ERT, even as short as a few months, worsens Pompe patients' motor and respiratory function., (© 2021 European Academy of Neurology.)
- Published
- 2022
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