Join a CureDuchenne event
CureDuchenne has a variety of fundraising events throughout the year. These events have helped us fund seven research projects that have gone to human clinical trials. Join us for the Champions to CureDuchenne Newport Beach Gala on March 29, 2014.
Moving research into practice
CureDuchenne’s accelerated push to move research from the lab into clinical trials could halt the progression of the disease and save the lives of thousands of Duchenne boys.
Together, we can CureDuchenne
Duchenne muscular dystrophy is a devastating muscle disease in children, affecting one in every 3,500 male births. Historically, Duchenne boys have not lived beyond their mid-20s. Those that do are faced with progressively weaker muscles and pulmonary and cardiac failure. The potential now exists, however, to make a difference in their quality of life.
Duchenne families have hope
Hear what Duchenne boys, their parents, and researchers have to say about the experience of living with Duchenne muscular dystrophy.
The eDystrophin database contains information from Duchenne and Becker muscular dystrophy patients searchable by mutation (deletions, duplications and substitutions).
Duchenne is a progressive, muscle-wasting disease.
It results from a defective gene responsible for producing the key muscle protein, dystrophin. Without dystrophin, cells easily become damaged and die, resulting in heart and breathing failure.
Click here for full image
What is CureDuchenne?
CureDuchenne is a national nonprofit that raises awareness and funds to find a cure for Duchenne muscular dystrophy. The funds we raise support the most promising research programs aimed at treating and curing the disease. For the first time ever there is hope to find a cure for this destructive disease affecting thousands of boys and their families.
Sign up for our newsletter
Exon skipping drugs will be used to transform Duchenne muscular dystrophy into a much milder disease as seen in Becker muscular dystrophy patients. eDystrophin is an online database that provides information about inframe mutations typically seen in patients with milder forms of the disease. The database has been constructed using patient records and is easy to use and searchable by mutation type or clinical phenotype.
The Latest from the Founder
Prosensa Outlines Next Steps for Drisapersen Program February 18, 2014
Leiden, February 18th , 2014 Dear Patient Group Representative, We hope this note finds you well. When we last communicated, we announced that Prosensa had regained all rights to drisapersen from GSK and retained the rights to the other DMD programs. Upon the announcement of the transfer of rights, GSK and Prosensa entered into a Read more »Continue reading