CureDuchenne, the leading nonprofit focused on finding a cure for Duchenne muscular dystrophy, launched a new website today atwww.CureDuchenne.org to further reflect its mission which focuses on improving and extending the lives of all those with Duchenne.
Duchenne muscular dystrophy is a fatal genetic disorder that causes muscle degeneration and typically effects boys. Currently there are nearly 15,000 boys in the U.S. battling this disease and 300,000 worldwide. Most boys with Duchenne lose their ability to walk by age 12 and do not survive beyond their mid-20s. As the disease progresses, the simplest of tasks become difficult, and in the later stages, heart and breathing muscles begin to fail. At this time, there is no cure for Duchenne.
“CureDuchenne continues to accelerate the cure, redefine care and build a community for all those impacted by Duchenne,” said Debra Miller, founder and CEO of CureDuchenne. “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.”
The new website provides resources and information on three core areas:
- Cure:With a goal of finding a cure for all genetic mutations of Duchenne, CureDuchenne is focused on funding the most promising and qualified preclinical and clinical research to save lives.
- Care:There are limited treatment options for Duchenne patients, and CureDuchenne provides a vital education resource for parents, caregivers and clinicians in order to help ensure loved ones have the best care.
- Community:CureDuchenne brings together families, caregivers and medical professionals to share experiences, advice, and personal stories, giving hope and support to the entire community.
The updated website allows viewers to find Duchenne-related resources ranging from research overviews to family stories about those affected with Duchenne. The website also includes information on free upcoming CureDuchenne Cares workshops; clinical trial information; fact sheets and infographics about Duchenne; details on exon skipping; Duchenne care topics such as physical therapy; and more.
The website comes at a pivotal time in continuing CureDuchenne’s progress towards finding a cure. This past year has been a victorious one in many ways, with the approval of the first ever drug to treat Duchenne; though Exondys51 is only effective for 13% of the Duchenne population, it’s a big step in the right direction. Another drug EMFLAZA, a steroid, was also approved for all boys with Duchenne.
CureDuchenne’s research portfolio is also highlighted on the new website in the CureDuchenne Ventures section. It includes 12 wide-ranging projects to help treat the whole disease. This pipeline of therapies comes from a diverse array of cutting edge technologies used to treat all aspects of Duchenne. CureDuchenne Ventures’ early funding helps Duchenne science attract future investments made by venture capital firms, biotech and pharmaceutical companies.
CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne also is the innovator bringing physical therapy and standard of care to local communities around the country through CureDuchenne Cares. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.