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ИНТЕРНЕТ-МАГАЗИН
Издательство Атмосфера


Телефон издательства

  
Нервные болезни
2020 / N 2

Болезнь Помпе с поздним началом: ферментозаместительная терапия, комплексное ведение и реабилитация пациентов, экспериментальные терапевтические подходы
С.А. Клюшников

References

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28. Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT; NEO1 Investigator Group. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: a phase 1, open-label, multicenter, multinational, ascending dose study. Neuromuscular Disorders 2019 Mar;29(3):167-86.
29. Zhu Y, Jiang JL, Gumlaw NK, Zhang J, Bercury SD, Ziegler RJ, Lee K, Kudo M, Canfield WM, Edmunds T, Jiang C, Mattaliano RJ, Cheng SH. Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease. Molecular Therapy 2009 Jun;17(6):954-63.
30. NIH. U.S. National Library of Medicine. Study to compare the efficacy and safety of enzyme replacement therapies avalglucosidase alfa and alglucosidase alfa administered every other week in patients with late-onset Pompe disease who have not been previously treated for Pompe disease (COMET). Available from: https://clinicaltrials.gov/ct2/show/NCT02782741 Accessed 2020 Jul 14.
31. Sanofi. Press releases. 2020 Jun 16. Sanofi’s investigational enzyme replacement therapy shows clinically meaningful improvement in critical manifestations of late-onset Pompe disease. Available from: https://www.pharmashots.com/press-releases/sanofis-investigational-enzyme-replacement-therapy-shows-clinically-meaningful-improvement-in-critical-manifestations-of-late-onset-pompe-disease Accessed 2020 Jul 14.
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33. Salabarria SM, Nair J, Clement N, Smith BK, Raben N, Fuller DD, Byrne BJ, Corti M. Advancements in AAV-mediated gene therapy for Pompe disease. Journal of Neuromuscular Diseases 2020;7(1):15-31.
34. Corti M, Liberati C, Smith BK, Lawson LA, Tuna IS, Conlon TJ, Coleman KE, Islam S, Herzog RW, Fuller DD, Collins SW, Byrne BJ. Safety of intradiaphragmatic delivery of adeno-associated virus-mediated alpha-glucosidase (rAAV1-CMV-hGAA) gene therapy in children affected by Pompe disease. Human Gene Therapy. Clinical Development 2017 Dec;28(4):208-18.

  

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