Fierce Biotech3h
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 ...
FourStatesHomepage.com1h
Cherokee Nation to assist individuals with rare diseases
With rare diseases affecting 1 in 10 Americans and over 7,000 known conditions, the Cherokee Nation is taking a significant ...
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.
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 ...
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.
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 ...