US FDA approves Alnylam's drug for rare heart condition
U.S. Food and Drug Administration approved Alnylam's drug to treat a rare and deadly heart disease on Thursday, allowing the ...
The Lancet4h
The artist and the scientist join forces for rare diseases
Art and science are disciplines that have historically co-existed as polarised entities. However, artists and scientists both ...
American Council on Science and Health6d
“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Pharmaceutical Commerce9h
PAP 2025: The Impact of Out-of-Pocket Costs on Patients with Rare Diseases
Jonathan James, CEO, Hope Charities, discusses the critical importance of maintaining access to life-saving medications for ...
Local family gets answers through IU Rare Disease Clinic
A local family said they now have important answers about their daughter thanks to the work of the Indiana University Rare ...
My son’s rare disease diagnosis spurred me to ditch Big Pharma for a biotech startup
Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.
The Lancet14h
Advancing policies for rare diseases in Europe
Fostering dialogue between neurologists, researchers, patient representatives, and policymakers is an important step towards ...
Mother with rare ALS touts 'miracle drug' that has stopped her disease
Raziel Green, 52, an active runner and mother of two, was diagnosed with a rare form of ALS in 2017. Since starting a ...
Prince Frederik of Luxembourg dies from rare disease
Prince Frederik of Luxembourg, the youngest son of Prince Robert of Luxembourg and Princess Julie of Nassau, has died after a ...
Managed Healthcare Executive2h
FDA Approves First-Ever Cell Therapy Treatment for Rare Progressive Eye Disease
Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells ...
The Citizen14h
Volunteers create awareness surrounding Rare Disease Day
Rare Disease Day was observed last Friday by a group of volunteers who took an hour out of their schedule to create awareness ...