After seven decades with no advances in the treatment of the genetic disorder classic congenital adrenal hyperplasia (CAH), ...

Qure's AMT-130 shows promising results for Huntington’s disease. Learn why QURE stock faces commercialization challenges ...

U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License ...

Huntington's disease (HD) is a rare, inherited disorder that causes the progressive breakdown of nerve cells in the brain. It's a condition that's passed down in families from generation to ...

The show was inspired by Joey Pipia’s diagnosis with Huntington’s disease, a rare genetic neurodegenerative disease. “The show is a chance to celebrate the joy of finding the ‘Wow!’ especially in life ...

Mutant HTT drives Huntington's, and animal data suggest lowering levels of the protein could improve outcomes in a rare disease that causes people to gradually lose the ability to walk ...

The show was inspired by Joey Pipia’s diagnosis with Huntington’s disease, a rare, genetic neurodegenerative disease. “The show is a chance to celebrate the joy of finding the ‘wow!’ especially in ...

Qure stock surges after FDA agrees on an accelerated approval pathway for its Huntington's disease treatment, AMT-130.

At the center of the deal is an experimental medicine, PTC518, which is currently being tested as a potential treatment for Huntington’s disease in ... target a range of rare illnesses, from ...

The drug, code-named "PTC518," is being tested in a mid-stage study for the rare disorder that affects ... opens new tab the development of its Huntington's disease drug last year after concerns ...