Opinion: California's Rare Disease Council Will Transform Care for People with ALS
For families affected by this rare, fatal disease, the journey is grueling — not only because of the diagnosis itself but due ...
Hosted on MSN2h
US FDA declines full approval for Intercept's liver disease drug
Ocaliva received the FDA's accelerated approval in 2016 to treat patients with primary biliary cholangitis, a rare disease ...
FiercePharma2h
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 ...
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 ...
Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Cherokees deserve help, treatment and hope – especially those who’ve long felt unseen by the health care system due to their ...
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 ...
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.” ...
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 ...
FiercePharma7d
Arrowhead targets rare disease patients, pushing message that hope is on the horizon
Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness ...
Minnesota Daily1d
Parents of children with rare diseases share their stories for Rare Disease Day
The University of Minnesota’s annual Rare Disease Day seminar this year, held at the end of February, explained how patients with rare diseases can utilize telehealth services nationally and ...
The American Journal of Managed Care4d
Rare Disease
Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence ...
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 ...