What began in the early 1990s as an informal gathering of seven European patient groups in the Italian port of…
Articles by Larry Luxner
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a…
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.
When school psychologist Wayne Rosenfield was 5 years old, he had an enlarged spleen. Doctors in his hometown of Springfield,…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second…
Germany’s Centogene is introducing a free smartphone app that lets people with Gaucher disease monitor their own progress. But…
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…