Resolution of the Expert Council “Clinical monitoring of patients with late-onset Pompe disease. Impact of next-generation enzyme replacement therapy on clinical outcomes in patients with late-onset Pompe disease”

1. Государственный реестр лекарственных средств. ОХЛП Нексвиазайм ЛП-№(002078)-(РГ-RU) от 31.03.2023. Доступно по: https://grls.rosminzdrav.ru/Grls_View_v2.aspx?routingGuid=62dc44e5-1640-47fc-befb-c4ae06957cb1.

2. Клинические рекомендации «Болезнь Помпе». Одобрено Научно-практическим советом Минздрава РФ, 2019. Доступно по: https://cr.minzdrav.gov.ru/recomend/317_1.

3. Diaz-Manera J., Kishnani P., Kushlaf H. et al. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Lancet Neurol 2021;20(12):1012–26.

4. Harlaar L., Hogrel J.-Y., Perniconi B. et al. Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease. Neurology 2019;93(19):e1756–e1767. DOI: 10.1212/WNL.0000000000008441

5. Schoser B., Laforêt P., Kruijshaar M. et al. 208th ENMC International Workshop: Formation of a European Network to develop a European data sharing model and treatment guidelines for Pompe disease Naarden, The Netherlands, 26–28 September 2014. Neuromuscul Disord 2015;25(8):674–8. DOI: 10.1016/j.nmd.2015.04.006

6. Straub V., Kishnani P., Díaz-Manera J. et al. Efficacy and safety of avalglucosidase alfa in participants with late-onset Pompe disease after 145 weeks of treatment during the COMET trial. ePoster presented at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS 27), Halifax, Nova Scotia, Canada, 11–15 October 2022.