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 ...
FiercePharma2d
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 ...
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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 ...
GEN13h
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.
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 ...
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 ...
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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 ...
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 ...
GlobalData on MSN1d
Intercept loses Ocaliva FDA full approval bid in rare liver disease
Intercept’s US president Vivek Devaraj said the pharma company still believes in the evidence supporting the liver disease ...
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 ...