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 ...

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

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

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 ...

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

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 ...

"Within 24 hours, we received $11,000. 38 hours later, it was close to $35,000," said Parker. "I have never in my life seen ...