Ilan Ganot, Co-Founder, President and CEO of Solid Biosciences discusses how his company views advocates and patients who have Duchenne muscular dystrophy (DMD). Mr. Ganot started Solid in 2013 to find treatments, and potentially a cure, for DMD, a disease that afflicts his son Eytani.
Kristen Wheeden, Director of Development for the American Porphyria Foundation, provides an overview of her foundation and it’s focus on physician education. The American Porphyria Foundation is also dedicated to the health and well-being of patient and families impacted by Porphyria.
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.