Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD) discusses some of the challenges facing the rare disease community, including the perception that drug pricing for orphan drugs is a driving force behind rising drug prices.
Becky Abbott, MPH, Manager of Treatment and Research at the National Foundation for Ectodermal Dysplasias (NFED) discuses her organization and ectodermal dysplasias (ED), a group of more than 180 disorders that affect the outer layer of tissue of the embryo (ectoderm) that helps make up the skin, sweat glands, hair, teeth, and nails.
Benjamin Forred, Translational Research Project Manager at Sanford Health in Sioux Falls, South Dakota, discusses Rare Disease Day and his organization which is one of the largest health systems in the United States, including research into rare diseases.
Monica Weldon of Bridge The GAP – SYNGAP talks about this rare neurological condition that is linked to autism.
Kyle Bryant is a rare disease advocate. and the founder/director of rideATAXIA for the Friedreich’s Ataxia Research Alliance (FARA). Kyle and his team produce family friendly bike rides across the country to empower those with Friedreich’s Ataxia and raise funds for research.