Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that ...

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.

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life.

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition ... and to so very many good friends and ...

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

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 ...

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 ...

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 ...

Consanguinity is common in multiple regions, particularly in the Middle East and Africa, and can have positive and negative consequences. A significant proportion of genetic diseases manifest as ...