AdvocacyLeading advocates and experts share their experiences
Dennis Jackman, Senior Vice President, Public Affairs at CSL Behring discusses rare disease drug pricing, patient involvement in the drug development process, and drug access.
Joe Pugliese discusses the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy services for persons with hemophilia and other bleeding disorders.
Patricia Weltin of Beyond the Diagnosis talks about Elhers-Danlos syndrome, a rare disease that afflicts two of her daughters.
David Pearce, PhD, of the Coordination of Rare Diseases at Sanford (CoRDS) talks about their patient advocacy conference held in Sioux Falls, SD.
Julie Raskin, Executive Director of Congenital Hyperinsulinism International (CHI) explains how congenital hyperinsulism is diagnosed and the latest research is underway to improve how it is diagnosed and understood.
Kristin Smedley is the President of Curing Retinal Blindness Foundation, a non-profit organization focused on finding treatments and cures for CRB1 retinal disease.
The Rare Disease Film Festival focuses exclusively on rare diseases will take place November 9-10, 2019 at the Mission Bay Conference Center in San Francisco.
Luke Rosen is the father of a daughter with KIF1A associated neurological disorder (KAND) and co-founder of the short-lived, but highly successful patient organization, KIF1A.org.
Neena Nizar, PhD, co-founder of the Jansen’s Foundation is the personification of the determination.
Patricia Welton has two daughters with Ehlers-Danlos syndrome. Her daughters had the disease for years before properly being diagnosed.