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

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 ...

By Dr. Kaushik Mandal Rare diseases very often remain in the shadows, as common people have limited awareness about them.

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 ...

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

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 ...

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 ...