Vyondys 53 (Golodirsen).

Three innovative new medicines for Duchenne muscular dystrophy (DMD) have been introduced to Hainan Boao Lecheng International Medical Tourism Pilot Zone by Boao Winhealth Rare Diseases Medical Center, local media outlets reported on Aug 30.

DMD is a rare disease inherited in an X-linked recessive pattern, which occurs primarily in males. The incidence rate of DMD among male infants is around one in 3,500.

DMD affects the muscles, leading to muscle deterioration that gets worse over time. Most of the patients may show symptoms from ages 3 to 5, lose walking ability at around 10 years old and may die of respiratory failure and heart failure in their 20s.

The three medicines, namely Exondys 51 (Eteplirsen), Vyondys 53 (Golodirsen) and Amondys 45 (Casimersen), are expected to help 70,000 to 90,000 domestic DMD patients who currently relying on hormonotherapy.

These three medicines have been approved by the Food and Drug Administration of the United States and entered the market in 2016, 2019 and 2021, respectively, and are antisense oligonucleotides for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exons 51, 53 and 45.

Boao Winhealth Rare Diseases Medical Center was jointly built by partners including the Boao Lecheng International Medical Tourism Pilot Zone Administration Bureau and Winhealth Pharma Group in April 2020, striving to bring the most advanced medicines for rare diseases to the domestic market.