ABOUT US
The DMD Project’s mission is to develop a groundbreaking cell-based therapy program designed to treat Duchenne muscular dystrophy (DMD), the most severe form of muscular dystrophy, which results in muscle degeneration and premature death. Our innovative technology utilizes a non-viral vector system carrying the full dystrophin gene designed to treat DMD. Working in collaboration, our translational approach to product development is based on the strong research foundation provided by our key partnerships.
Individuals with DMD lack or produce insufficient dystrophin, leading to muscle cell damage and replacement with fat and fibrotic tissue over time. However, innovative therapies targeting the underlying genetic cause offer hope for improved outcomes.
These advancements hold promise for enhancing the prognosis and quality of life for individuals affected by DMD, offering newfound optimism for managing this debilitating condition.
1 in 3500 to 5000 males
Affected by DMD
Ages 3-5
Symptoms will first appear
Age 12
Many patients will need wheelchair assistance.
$63.5B
Market evaluation by 2030
https://www.coherentmarketinsights.com
