An international, Phase III clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare ...

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 ...

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

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

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

In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...

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

Intercept’s US president Vivek Devaraj said the pharma company still believes in the evidence supporting the liver disease ...

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

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 ...