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

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

Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...

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

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

Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness ...

About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare Disease Summit ...

Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most ...

In a report released today, Andrew Fein from H.C. Wainwright reiterated a Buy rating on BioCryst (BCRX – Research Report), with a price ...

While PH Awareness Month is a great opportunity to connect and educate, rare disease advocacy is columnist Jen Cueva's passion all year.

On October 4, 2024, a US House version of the revised Promising Pathway Act (PPA) 2.0 was introduced, sponsored by Rep. Bruce Westerman (R-AR).

Patient engagement is a necessity when performing health technology assessment to help better assess and identify the outcomes of a rare disease.