Duchenne Muscular Dystrophy
Striving for Better Patient Outcomes
Duchenne muscular dystrophy, one of the various forms of muscular dystrophy, is a rare genetic disease that affects boys almost exclusively and causes progressive weakness.1 Patients typically lose the ability to walk when they reach 8 to 12 years of age, and die as a result of respiratory or cardiac failure.1 Before treatments were available, children with the disease typically died in their teens.2 As a result of steroid use and supportive respiratory therapies, they now live well into their 20s,3 but more must be done to allow these patients to live a fuller life.
This condition is a priority for Pfizer, and our researchers are developing an investigational gene therapy that is currently being evaluated in a clinical trial. In addition, several companies are working on treatments using gene therapy and gene repair.