More than 30 million people in the United States are affected by rare diseases, many of which are life-threatening conditions without available treatments. On Thursday, Feb. 29 at 2:00 p.m. ET, join Washington Post Live for conversations about the ongoing challenges around rare diseases, the paucity of medical data and the role of citizens in advancing research and solutions.
The Akari Foundation, All Wheels Up, Child Neurology Foundation, Cure CMD, Cure Rare Disease, Cure VCP Disease, and Christopher & Dana Reeve Foundation are.
/PRNewswire/ ResearchAndMarkets.com announces the 4th Annual Orphan Drugs and Rare Diseases Conference, scheduled for October 9-10 in London, UK. This.
The lone volunteer in a gene-editing study targeting a rare form of Duchenne muscular dystrophy likely died after having a reaction to the virus that delivered the therapy in his body, researchers concluded in an early study. Terry Horgan, 27, of Montour Falls, New York, died last year during one of the first tests of […]