The IV All-Russian Conference “Duchenne Muscular Dystrophy: Time Matters”, organized by the Gordey Charitable Foundation, focused on early diagnosis and therapy to preserve motor functions and slow disease progression. A session on “Wiltolarsen – a registered therapy for Duchenne muscular dystrophy” was held at the conference, supported by R-Pharm Group of Companies.
Viltolarsen is designed to treat Duchenne muscular dystrophy in patients with a confirmed mutation in the dystrophin protein gene that allows for exon 53 skipping. Developed by the Japanese pharmaceutical company Nippon Shinyaku Co., Ltd., viltolarsen was added to the Circle of Good Foundation’s list of essential medications in 2022 to support children facing severe, life-threatening conditions, including rare diseases. In 2024, R-Pharm, under a licensing agreement with Nippon Shinyaku, successfully registered viltolarsen for use in Russia.
