Fierce Biotech2h
AstraZeneca's $800M rare disease bet hits primary phase 3 goal, but competitive strength still unclear
A phase 3 trial of AstraZeneca’s $800 million rare disease candidate has met its primary endpoint. The update keeps ...
American Council on Science and Health3d
“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
For kids with extremely rare diseases, this promising technology provides a lifeline | Opinion
Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that ...
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 ...
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 ...
Live Science on MSN4d
POLG diseases: Rare genetic conditions that starve cells of energy and afflicted the late Prince of Luxembourg
POLG-related diseases disrupt the function of the mitochondria, or "powerhouses" of the cell — starving them of energy.
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.
Rare disease patients need Trump’s attention to make America truly healthy again
The Trump administration should work with Congress to advance these two rare disease priorities to help make America healthy ...
What Is Hantavirus, the Rare Disease That Killed Betsy Arakawa?
Ms. Arakawa, the wife of the actor Gene Hackman, died from the effects of a disease often caused by contact with droppings ...
A Young Prince of Luxembourg Dies of a Rare Genetic Disease
Prince Frederik, 22, was a founder and the creative director of a foundation that raised awareness of POLG disorder.
Woman diagnosed with rare disease after 'excited' dog accident
An ex-RAF nurse has said that the detection of her rare disease was all thanks to an accidental collision with her family's ...
FSU's Rare Disease Day spotlights promising research for families
Rare Disease Day provided a forum for university and state leaders to lay out the future plans and share the work already ...