THE CURE IS WITHIN REACH
20 years ago, we set out to get Duchenne research out of labs and into clinics. Our next 3-5 years will be focused on translating these experimental drugs into approved therapies leading to curative treatments.
- Funded 17 research projects that have advanced to human clinical trials
- Funded multiple projects aimed at overcoming the limitations of gene therapy
- Contributed early funding to the first FDA-approved Duchenne drug
- Pioneered the first and only Duchenne physical and occupational therapist certification program
- Successfully implemented a hospital-based pilot to screen newborns for Duchenne
- Created an innovative biobank and data registry, facilitating data sharing between patients and researchers
We know you will want to be a part of this countdown to a cure and ask you to please lend your support.