Duchenne Population Potentially Amenable to Exon Skipping
Exon skipping is a potential treatment approach to correct and restore production of dystophin. For specific genetic mutations it allows the body to make a shorter, usable dystophin. Exon skipping is not a cure for Duchenne, but it may make the effects less severe.
Get the information you need to provide better care for those with Duchenne. The CureDuchenne Cares mission is to increase the health and wellbeing of patients living with Duchenne through educating parents, caregivers, clinicians, and physical therapists on best practices for Duchenne management. This program also keeps families informed on the latest scientific develipments in Duchenne research, including status and participation in clinical trials.
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.
Duplication Mutation Therapy for Duchenne
CureDuchenne has provided the lead funding for Dr. Flanigan's duplication research and is committed to helping bring this therapy into trials. We need your support in raising $710,000 to continue this important research.
Help Advance Research to Treat Heart Disease
CureDuchenne Ventures LLC is investing $1 million in Capricor Therapeutics to advance promising research to treat disease associated with Duchenne muscular dystrophy.
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.
The Latest from the Founder
Pfizer Clinical Trial Update December 8, 2016
CureDuchenne is pleased to share the following updates from Pfizer pertaining to the on-going multicenter Phase II clinical trial of the investigational compound, domagrozumab (PF-06252616) in boys with Duchenne muscular dystrophy (DMD). Domagrozumab is an experimental, infused, anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it Read more »Continue reading