Jaguar Health Inc. surged as investors cheered positive clinical trial progress, and its stocks have been trading up by 34.25 ...

The family of a 13-year-old boy from Long Beach is asking for help as he remains hospitalized with a rare disease linked to ...

When the aging brain misfires, the results can be astonishing and a little alarming.

Krystal Biotech, Inc. (NASDAQ:KRYS) has something most biotech companies spend years trying to achieve: a drug that works, is approved, and is already generating meaningful revenue. VYJUVEK, a gene ...

A California boy who was once a healthy, soccer-loving teenager is now fighting to regain the use of his legs after a seemingly harmless mosquito bite triggered a devastating medical nightmare. Joseph ...

At her confirmation hearing, Dr. Heidi Overton, President Donald Trump’s nominee to lead the Food and Drug Administration, ...

The first global assessment of rare neurological diseases in children finds rising prevalence and disability from 1990 to 2021, declining mortality, and a projected 11.83 percent increase in cases by ...

The number of treatments for rare genetic disorders is expected to increase sharply in the next several years.

DANCING with the Stars judge Carrie Ann Inaba has spoken out about living with a rare medical condition. While the TV ...

“I just want people to see Luca as a little boy first,” Mariah George tells PEOPLE. “His diagnosis is a part of his story, ...

A Palestinian family case series shows four girls with the same homozygous WFS1 mutation developing strikingly different ...

The appendix grant is a specimen of exactly that mechanism, not the catalyst itself. The dollar amount is immaterial; what is ...