Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA-approved drug indicated for the rare lipid storage disease and autosomal recessive genetic ...

Mirum Pharmaceuticals' Ctexli wins FDA approval for CTX, a rare lipid storage disease. The company projects 2025 global sales of $420–$435 million.

This rare genetic disorder was just treated in the womb for the first time ever - Without treatment, those with the more ...

Lyric Mariah Heard spoke with "Good Morning America" about her life as a model born with a rare birth defect and using humor ...

The White-bellied Heron (WBH), the world's second-largest heron, is a symbolic bird for the people of Bhutan and also a ...

33.This is Mary Ann Bevan, a widow who was given the title of "World's Ugliest Woman" in 1920: After the death of her husband and being diagnosed with a rare disease, Mary Ann joined circus ...

Genetic testing helps prevent the passing of genetic deformities. The Albino Poodle is a rare genetic wonder, boasting white fur, pink noses, and light blue or red eyes. This unique appearance results ...

This type is very rare and often occurs due to an absent part of the X chromosome. Other Turner syndrome abnormalities include: X chromosome abnormalities. These can happen when your cells have ...

The U.S. has imposed a 10% tariff on all Chinese goods, including rare earths, and China has retaliated with export controls on critical minerals. Conflict in Myanmar has disrupted the supply of ...

Now add this to the mix: U.S. President Donald Trump wants to condition future U.S. aid to Ukraine on getting more access to the country's valuable "rare earth" minerals -- minerals that are in ...