Opinion: California’s Rare Disease Council Will Transform Care for People with ALS
California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...
FiercePharma1h
FDA rejects Intercept's ask for Ocaliva full nod in rare liver disease, but accelerated approval remains
In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...
pharmaphorum4d
Patient Recruitment for Rare Disease Trials Summit
Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical ...
EurekAlert!1d
Stand Up to Cancer collaborates with Johnson & Johnson to explore targeted therapies for rare disease linked to blood cancers
Stand Up To Cancer® (SU2C) today announced a collaboration with Johnson & Johnson aimed at supporting research testing a ...
Developing tailor-made treatments for ultra-rare disease patients: Research provides a roadmap
In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...
STAT8d
My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?
Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...
Native News Online2dOpinion
Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Guest Opinion. About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare ...
Decades-Long Medical Mystery Solved: New Rare Genetic Disorder Identified
Researchers at Washington University have discovered a new genetic disorder affecting protein folding, offering potential new treatment avenues for rare brain conditions. When most people feeling ...
Science News3d
A common drug may help treat a rare genetic disease
Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...
Ottawa boy with rare disease gifted Taylor Swift tickets auctioned off on his behalf
An Ottawa boy with a rare disease will be getting the chance to see Taylor Swift in concert after the winner and organizers ...
Tahlequah Daily Press4d
Cherokee Nation to host Rare Disease Summit in Tahlequah Nov. 21
The Cherokee Nation is hosting a Rare Disease Summit Nov. 21 to address the unique challenges facing tribal citizens with rare medical conditions.