Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that could accelerate the discovery of new treatments. | Opinion ...

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more ...

5 CTX is an extremely rare genetic metabolic disorder that is caused by a mutation in the CYP27A1 gene, leading to reduced bile acid production in the liver and an inability to break down ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

Prince Frederik of Luxembourg died on March 1, 2025, at the age of 22 after a prolonged battle with POLG mitochondrial disease — a rare genetic disorder that causes progressive multi-organ failure.

He passed away on March 1 from POLG Mitochondrial disease, a rare genetic disorder that affects ... “It is with a very heavy heart that my wife and I would like to inform you of the passing ...

Luxembourg’s Prince Frederil raised awareness of PolG disease, a rare health condition, he battled before his death at 22 ...

It’s a rare genetic disorder that affects the nervous system and can be fatal.“We were told that it has a really high false positive rate, and it's extremely rare, and really not to worry ...

a rare and fatal genetic disorder affecting the central nervous system. On the very day Noa was diagnosed, an experimental gene therapy and clinical trial were posted to treat Canavan disease.

"Getting the correct diagnosis can be the first step to decreasing the burden of these extremely ... rare and life-threatening diseases.” The Alnylam Act ® program offers no-cost, confidential ...