In this first in a series of podcasts focused on rare disease patient advocates, we speak to Debra Miller, president and founder of Cure Duchenne. When Miller’s five-year-old son was diagnosed with Duchenne Muscular Dystrophy, she faced grim news. The progressive muscle loss caused by the disease typically leaves these children wheelchair-bound by age 10 and robs them of their lives by 20.
As she and her husband explored treatment options and ways they could become to raise money for research, they found that existing organizations were often focused on palliative care and focused on academic research rather than working with industry.
Miller and her husband launched Cure Duchenne to help fund promising research at biotech companies in the hopes of seeing these therapies developed and commercialized. We spoke to Miller about her son’s story, new approaches to treating Duchenne, and the problems of funding cutting-edge research.