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 ...
STAT10d
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 ...
FiercePharma1d
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 ...
EurekAlert!3d
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 ...
News on 61d
Tulsa Mechanic, Community Rally To Give 21-Year-Old With Rare Disease Gift Of Adventure
In Tulsa, mechanic Matt Parker rallied the community to support 21-year-old Corbin Norcom, who has a fatal disease, by ...
Warrington Wolves CEO reveals fear after being struck down by rare disease
Karl Fitzpatrick has described his frightening ordeal after being diagnosed with potentially life-threatening Guillain–Barré ...
GEN16h
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
to use its artificial intelligence to identify novel rare disease indications for a discontinued drug asset. Under the terms of the agreement, Sanofi will provide data related to the unnamed drug ...
Science News5d
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.” ...
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 ...
Minnesota Daily3d
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 ...
Actuate announces FDA rare pediatric disease designation to elraglusib
Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...
The American Journal of Managed Care6d
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 ...