Mother with rare ALS touts ‘miracle drug’ that has stopped her disease
Eight years after receiving a life-shattering diagnosis, a New Jersey mother credits an “amazing” new drug for stopping her ...
Threat to son's treatment for rare disease 'agony'
A mother has spoken about her "painful" experience over the potential end to her son's life-enhancing treatment for a rare ...
My son’s rare disease diagnosis spurred me to ditch Big Pharma for a biotech startup
Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.
First responders continue to help young girl with rare disease collecting patches to ...
ROCKY HILL, Conn. (WFSB) - First responders continue to meet the request for a young girl with a rare disease. Earlier this ...
What is PolG? The rare genetic disease that ended a young prince's life
Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more ...
pharmaphorum5 天
Why is rare disease funding rare?
Oliver Timmis explores the problems associated with securing funding for rare disease research and questions why more funding isn't available in this space. It's difficult to research rare diseases.
E! Online4 小时
Gene Hackman and Betsy Arakawa’s Friends Share Rare Insight Into Decades-Long Marriage
After Gene Hackman and Betsy Arakawa were found dead in their New Mexico home, their friends opened up about the couple’s ...
Sialidosis therapy: Kiwi siblings fundraise for injection against disease that killed brother
Two Kiwi siblings with the same rare genetic condition that took their brother’s life are fundraising for a groundbreaking ...
Prince Frederik of Luxembourg dies from rare disease
Prince Frederik of Luxembourg, the youngest son of Prince Robert of Luxembourg and Princess Julie of Nassau, has died after a ...
pharmaphorum1 天
Rare Disease R&D: one step forward, but a long road ahead
There are approximately 7,000 different types of rare disease and researchers estimate that there are more than 300 million people worldwide living with such a condition. When taken ...
Prince Frederik of Luxembourg dies at 22 from rare mitochondrial disease
The prince had PolG disease, a genetic mitochondrial disorder that prevents the body's cells from producing adequate energy.