AdvocacyLeading advocates and experts share their experiences
- Solid Biosciences and the Role of Advocates
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.
- American Porphyria Foundation’s Focus on Physician Education
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.
- DMD Research Activity and the Role of the Advocate
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: NORD’s Focus on the Rare Disease Patient
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.
- Mucopolysaccharidosis (MPS) Resource Center
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).
- Clinical Research and Finding a Cure for Duchenne Muscular Dystrophy
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.
- Choosing a Clinical Trial for Duchenne Muscular Dystrophy Patients
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.
- Dup15q Alliance and Syndrome
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: American Porphyria Foundation and Disease Overview
Kristen Wheeden, Director of Development for the American Porphyria Foundation, provides an overview of her foundation and this rare disease.
- Debra Miller Provides an Overview of CureDuchenne
Debra Miller is the founder and CEO of CureDuchenne, and in this video she provides an overview of the organization.