CureDuchenne, the Orange County non-profit focused on finding a cure for Duchenne muscular dystrophy (DMD) is one step closer to that goal through the work of Dr. Jeff Chamberlain.
“The focus of the trial will be on how the patient's immune system will respond to the gene delivery”
Dr. Chamberlain, of the University of Washington, in partnership with Dr. Stephen Tapscott, of the Fred Hutchinson Cancer Research Center, has identified a promising gene therapy that could lead to a treatment for DMD. The method delivers a new gene to the body's muscles to replace the defective, disease-causing gene.
As the leading genetic killer of young boys, DMD affects more than 1 in 3,500 boys worldwide and is the most common and lethal form of Muscular Dystrophy. Boys with DMD are usually diagnosed by the age of five, in a wheelchair by age 12, and may be completely paralyzed by their late teens. Historically, most patients with DMD do not live to see adulthood. Duchenne can occur in any family, from any race and from any background.
Debra and Paul Miller, co-founders of CureDuchenne will allocate the $250,000 raised during last weekend's 2010 Climb to CureDuchenne: Pick Your Peak, where teams across the country summitted mountains from coast to coast to raise awareness for DMD, along with the $165,000 raised by Mack and Sally Brown of Austin, TX during CureDuchenne's annual "Dealing for Duchenne" fundraiser, to fund Drs. Chamberlain and Tapscott's pre-clinical research.