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.
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 3rd Annual Getzlaf Golf Shootout on September 7 and September 8, 2013.
The eDystrophin database contains information from Duchenne and Becker muscular dystrophy patients searchable by mutation (deletions, duplications and substitutions).
Help us raise funds to find a cure for Duchenne. CureDuchenne can help you turn your ideas and passions into funding that will help accelerate Duchenne research.
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.
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.
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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.
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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.
Join Ryan Getzlaf, captain of the Anaheim Ducks, for the Getzlaf Golf Shootout – a two-day charity event, including a reception and a golf tournament – that brings together athletes, celebrities and community leaders, all teaming up in support of CureDuchenne.Register Now!
The Latest from the Founder
Summary of GSK – Drisapersen Webinar May 6, 2013
DRISAPERSEN UPDATE: results from a randomized, double blind, placebo-controlled Phase II clinical trial Webinar with GlaxoSmithKline took place on Monday 6th May 2013, 9:00 am PDT. The webinar was to inform and update the Duchenne community on the Phase II clinical trial results for GSK2402968 (drisapersen) (DMD study 114117). Drisapersen is a 2’-O-methyl-phosphothioate oligo designed Read more »Continue reading