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.
Mucopolysaccharidoses (MPSs) are a group of genetic lysosomal disorders in which persons have low levels of specific enzymes that leads to an abnormal accumulation of complex carbohydrates (mucopolysaccharides or glycosaminoglycans).
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.
Debra Miller is the founder and CEO of CureDuchenne, and in this video she provides an overview of the organization.