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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.
7-year-old in Hamilton has rare condition with $300K annual treatment her family can't afford
Cena Hermus, 7, was two years old when she was diagnosed with a rare, life-threatening disorder that causes severe obesity.
Polar Plunge in honor of Staten Islander with rare skin disease planned for this month
STATEN ISLAND, N.Y. — A fundraising Polar Plunge to honor John Hudson Dilgen will take place this month in South Beach to ...
Primary school pupils brighten up Rare Disease Day with bold outfits
Pupils at Bridport Primary School put the spotlight on rare diseases by dressing in a riot of colour and learning about the challenges faced by those affected. As part of Rare Disease Day, children at ...
Director Who Worked With Prince Frederik Says it Was ‘Painful’ to Watch Him Struggle Before His Death
Filmmaker Mei Fa Tan reflected on her experience working with Luxembourg’s Prince Frederik before his death at the age of 22.
Prince Frederik of Luxembourg’s final days: Filmmaker recalls 22-year-old's battle with rare disease
Two weeks after Prince Frederik of Luxembourg died from POLG, a documentary filmmaker has revealed that the 22-year-old ...
Healio58m
Q&A: ‘An exciting breakthrough’: Gene transfer therapy promising in Danon disease
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
Batten Disease: Parents fight for costly drug for children's rare disease to remain on NHS
Oliver and Amelia Carroll have Batten Disease - an incurable genetic disorder that causes rapid decline in a child's ability to walk, talk and see. | ITV News Granada ...
The American Journal of Managed Care1h
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access
The study found that just 20% of trials submitted to both the FDA and European Medicines Agency had matching evidence.
Terrifying and painful new skin disease ravages young father, with doctors warning it will kill him
A 39-year-old father is suffering from a horrifically painful skin disease that doctors can't cure or even definitively ...
NC Biotech33m
UNC KickStart Venture Services celebrates awards to university startups
“The KickStart Grant Awards help de-risk these technologies by establishing a collaboration agreement between the startup ...
E! Online43m
Gene Hackman Case: How Surviving Dogs Helped Paramedics Find His Body
After paramedics found Gene Hackman’s wife Betsy Arakawa dead in the bathroom of their Santa Fe home, the couple’s dogs led authorities to the actor’s body.