Journal
EUROPEAN JOURNAL OF NEUROLOGY
Volume 29, Issue 4, Pages 1181-1186Publisher
WILEY
DOI: 10.1111/ene.15222
Keywords
alglucosidase alfa; enzyme therapy; stopping; suspension; treatment management
Categories
Funding
- Sanofi Genzyme
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This French study shows that even a few months of interruption in ERT can worsen the motor and respiratory function of patients with Pompe disease.
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.
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