CureDuchenne and the DMD community have taken heart from two recent Federal Drug Administration approvals. The initial one, in September 2016, was for the first-ever drug to treat Duchenne, Exondys51 (eteplirsen). The downside of that announcement is that Exondys51 is effective in only 13 percent of the DMD population.
To help the DMC community continue progressing and advance toward a cure, the reconstituted website focuses on providing timely resources and information in three main areas:
- Cure – to find a cure for all genetic mutations of DMD, the nonprofit is working on funding the most promising and qualified preclinical and clinical research.
- Care – to provide vital education resources for patients, caregivers and clinicians to help ensure loved ones get the care they need.
- Community – to bring together families, caregivers and medical professionals to share experiences, tips, advice and personal stories to support the DMD community.
The new-look site offers DMD-related resources, CureDuchenne Cares workshops, clinical trial information, fact sheets and statistics about DMD, details about the gene-based treatment approach called exon skipping, and information about physical therapy and other topics. Check it out!
CureDuchenne was founded in 2003 to improve and extend the lives of those living with DMD, a disease estimated to affect over 300,000 boys worldwide.
The organization is involved in 12 wide-ranging projects to treat the disease. CureDuchenne Ventures is supporting the effort. It helps find venture capital firms, biotechnology companies and pharmaceutical companies interested in investing in DMD scientific projects.
CureDuchenne has helped advance nine research projects into human trials with the help of donations and lots of volunteers’ time.
“CureDuchenne continues to accelerate the cure, redefine care and build a community for all those impacted by Duchenne,” Debra Miller, founder and chief executive officer of CureDuchenne, said in a press release. “We are dedicated to finding a cure for this devastating disease. We hope that with the help of the new website, individuals will be able to learn more about Duchenne and take action to help find a cure.”