Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo…
Articles by Larry Luxner
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…
Two organizations that advocate for people with Prader-Willi syndrome have teamed up with Boston-based Zafgen to conduct an unprecedented…
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…
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country.
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves…