Russian Journal of Child NeurologyRussian Journal of Child NeurologyРусский журнал детской неврологии2073-88032412-9178Publishing House ABV Press52010.17650/2073-8803-2025-20-2-40-51REVIEWS AND LECTURESОБЗОРЫ И ЛЕКЦИИVamorolone – a corticosteroid approved by global regulators for the treatment of Duchenne muscular dystrophy. Literature reviewВаморолон – одобренный мировыми регуляторами глюкокортикостероид для терапии прогрессирующей мышечной дистрофии Дюшенна. Обзор литературыhttps://orcid.org/0000-0003-2635-2752VlodavetsD. V.ВлодавецД. В.
Russian Federation

Dmitriy Vladimirovich Vlodavets 

2 Taldomskaya St., Moscow 125412

Дмитрий Владимирович Влодавец

125412 Москва, ул. Талдомская, 2

mityaus@gmail.comhttps://orcid.org/0000-0002-8876-7462ArtemyevaS. B.АртемьеваС. Б.
Russian Federation

2 Taldomskaya St., Moscow 125412

125412 Москва, ул. Талдомская, 2

https://orcid.org/0000-0002-4858-9251TozliyanE. V.ТозлиянЕ. В.
Russian Federation

2 Taldomskaya St., Moscow 125412

125412 Москва, ул. Талдомская, 2

https://orcid.org/0009-0001-2218-9874ShulchenkoA. K.ШульченкоА. К.
Russian Federation

Build. 1, 96 Prospekt Vernadskogo, Moscow 119571

119571 Москва, проспект Вернадского, 96, корп. 1

Scientific Research Clinical Institute of Pediatrics and Pediatrics Surgery named after Acad.Yu.E. Veltischev, N.I. Pirogov Russian National Research Medical University, Ministry of Health of RussiaНаучно-исследовательский клинический институт педиатрии и детской хирургии им. акад. Ю.Е. Вельтищева ФГАОУ ВО «Российский национальный исследовательский медицинский университет им. Н.И. Пирогова» Минздрава РоссииA.P. Nelyubin Institute of Pharmacy, I.M. Sechenov First Moscow State Medical University, Ministry of Health of RussiaИнститут фармации им. А.П. Нелюбина ФГАОУ ВО Первый Московский государственный медицинский университет им. И.М. Сеченова Минздрава Россииru040820252025202405131072025Received:31072025Published:04082025Copyright ©; 2025, ABV-PressCopyright ©; 2025, АБВ-пресс2025ABV-PressАБВ-прессhttps://rjdn.abvpress.ru/jour/about/editorialPolicieshttps://rjdn.abvpress.ru/jour/article/view/520

Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease that manifests in early childhood and progresses with age, leading to loss of mobility and respiratory and cardiac failure. The globally recognized gold standard for DMD treatment is corticosteroids. However, until recently, only deflazacort (Emflaza) had received approval from the U.S. Food and Drug Administration (FDA) for the indication of DMD. All other drugs in this group were used off-label. At the end of 2023, the FDA, followed by the European Medicines Agency (EMA), registered another drug from the corticosteroid group – vamorolone (Agamree). In 2024, vamorolone received approval in the United Kingdom and China. Thus, all leading global regulators currently recognize vamorolone as a corticosteroids for the treatment of DMD.

Прогрессирующая мышечная дистрофия (ПМД) Дюшенна – X-сцепленное наследственное заболевание, проявляющееся в раннем детстве и прогрессирующее с возрастом, приводящее к потере возможности двигаться, дыхательной и сердечной недостаточности. Общепризнанным мировым «золотым стандартом» лечения ПМД Дюшенна являются глюкокортикостероиды. Однако до последнего времени только дефлазакорт (Эмфлаза®) имел одобрение Управления по санитарному надзору за качеством пищевых продуктов и медикаментов США (Food and Drug Administration, FDA) по показанию «ПМД Дюшенна». Все остальные препараты этой группы применялись off-label. В конце 2023 г. FDA, а затем и Европейское агентство лекарственных средств (European Medicines Agency, ЕМА) зарегистрировали еще один препарат из группы глюкокортикостероидов – ваморолон (Агамри®, от англ. Agamree®). А в 2024 г. ваморолон получил одобрение в Великобритании и Китае. Таким образом, ваморолон в настоящее время является признанным всеми ведущими мировыми регуляторами препаратом из группы глюкокортикостероидов для терапии ПМД Дюшенна.

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