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  • [Reporter's View] Concerns about new drugs for rare diseases
  • by Son, Hyung Min | translator Hong, Ji Yeon | Mar 7, 2025 05:56am
Duchenne muscular dystrophy is a rare muscular disorder caused by the lack of dystrophin, primarily affecting boys. The symptoms typically begin before the age of 3 and rapidly deteriorate, eventually leading to the loss of ability to walk before the age of 10 and above.

To date, various genetic targeted therapies for Duchenne muscular dystrophy won FDA approval, including U.S.-based Sarepta Therapeutics' 'Amondys 45,' 'Exondys 51,' 'Vyondys 53,' and 'Elevidys,' the American subsidiary of Japan's Nippon Shinyaku NS Pharma's 'Viltepso,' and Italfarmaco's 'Duvyzat.'

Since these new drugs have not been introduced to South Korea, so patients with Duchenne muscular dystrophy are relying on steroids.

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