For families affected by this rare, fatal disease, the journey is grueling — not only because of the diagnosis itself but due ...

Both events are taking place simultaneously 19–20 November in Brussels, Belgium. As interest in rare disease drug development ...

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 ...

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 ...

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

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

Karl Fitzpatrick has described his frightening ordeal after being diagnosed with potentially life-threatening Guillain–Barré ...

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 ...