When I arrived through the gates at the Barretstown camp, I was eager to meet my campers and fellow group of volunteers, caras, Gaelic for friend. What I didn’t know is what a lasting impact this camp, set in Ballymore Eustace, Ireland, would have on my life. read more >
Obstacles in rare disease research and orphan drug development continue to include low patient numbers, difficult recruitment, and insufficient scientific and medical knowledge such as lack of natural history data and known, relevant endpoints. Until now, collection of such information has resided primarily with clinical experts and drug development companies. But the recognition of patient registries as key determinants in accelerating research and drug development progress has propelled significant interest from patient organizations over the last decade. Today, read more >
From Left to Right: Mary Dunkle- NORD, Christy Greeley- Cystinosis Research Network, Ron Bartek-Friedrich’s Ataxia Research Alliance, Jeff Wuchich- Alternating Hemiplegia of Childhood Foundation, Mirta Avila Santos, American Behcet’s Disease Association (ABDA), Sandy McElgunn- ABDA, Megan O’Boyle- Phelan-McDermid Syndrome Foundation
Within the first hour of the Annual DIA-NORD U.S. Conference on Rare Diseases & Orphan Products, NORD and DIA hosted a panel of six patient advocates to help remind everyone that patients are at the heart of everything we will be talking about in the next few days.