Debra Miller, founder of CureDuchenne, discusses research activity in Duchenne muscular dystrophy (DMD) and the role of the advocate in this rare disease.
Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses his organization’s focus on the rare disease patient.
Debra Miller is the founder and CEO of CureDuchenne, and in this video she discusses some of the current clinical research in Duchenne muscular dystrophy (DMD) and the hope of a possible cure.
Debra Miller, founder of CureDuchenne, discusses some of the decisions that Duchenne muscular dystrophy (DMD) families and patients need to consider when considering a clinical trial.
Vanessa Vogel-Farley, Executive Director of Dup15q Alliance discusses her organization, which provides family support and promotes awareness, research and targeted treatments for chromosome 15q11.2-13.1 duplication syndrome.
Kristen Wheeden, Director of Development for the American Porphyria Foundation, provides an overview of her foundation and this rare disease.