Rare diseases have become an attractive target for pharmaceutical companies both on the ASX and internationally.

One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...

We designed our dream home in Mexico for $270,000. It was cheaper than buying in the US but came with extra costs we didn't ...

Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.

Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...

TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...

Travere’s stock price climbed by 14% following the announcement that it will seek an FDA label expansion for Filspari.

Charleigh Pollock, 9, is being treated for a terminal neurological disorder known as Batten disease with twice-monthly infusions of a drug that costs about $1 million per year.

Barcelona-based sales agency Jambika Docs has acquired the documentary feature “Noah” ahead of the European Film Market in ...

In a landmark discovery, researchers applied a novel AI model called the max-logistic intelligence classifier to analyze ...

The event featured not only Austin Laurette, who is "the unbreakable boy," but also some of the stars of the movie, Zachary ...