Larry Luxner

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have.

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails…

Hemophiliacs in Europe have never had so many reasons to feel hopeful about the future, thanks to significant recent scientific…

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the…