Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...

In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...

Stand Up To Cancer® (SU2C) today announced a collaboration with Johnson & Johnson aimed at supporting research testing a ...

Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...

Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.

In Tulsa, mechanic Matt Parker rallied the community to support 21-year-old Corbin Norcom, who has a fatal disease, by ...

Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical ...

Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness ...

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 ...

Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...

An Ottawa boy with a rare disease will be getting the chance to see Taylor Swift in concert after the winner and organizers ...

While PH Awareness Month is a great opportunity to connect and educate, rare disease advocacy is columnist Jen Cueva's passion all year.