California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...

Cherokees deserve help, treatment and hope – especially those who’ve long felt unseen by the health care system due to their ...

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 ...

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

“Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with EWS and recognizes elraglusib’s transformative potential,” said ...

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

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

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

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 ...

Profit after tax came in ₹38.4 crore in Q2 of FY25, a recovery from a net loss of ₹11.8 crore in the first quarter of this ...

Elraglusib is undergoing investigation in an open-label, multicenter, phase 1/2 trial for the treatment of patients with ...