Treatments that target a fragment of the mutant protein that causes Huntington's disease might be more effective than treatments, now in clinical ...

The Alliance was born from a classic D.C. moment: three Illinois advocates, standing in a hallway, waiting to meet with Senator Dick Durbin. That is where Wayne Galasek of Oak Park, Doug Butchart of ...

The MarketWatch News Department was not involved in the creation of this content. Industry veteran brings more than 30 years of experience launching therapies for neurological diseases, including ...

This will be Prasad’s second exit from the FDA during the current Trump administration. In July, he resigned amid his controversial handling of a gene therapy treatment for Duchenne muscular dystrophy ...

"The stories are so outrageous," Senator Ron Johnson said. "It just appears that they’re looking for excuses to say no." ...

UniQure’s AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain. ・The FDA recently said that data ...

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new ...

Investors are scrutinizing the prospects for some drugs in the pipeline following a string of recent rejections from the U.S.

The FDA’s dispute with biotech firm uniQure over Huntington’s gene therapy highlights growing tensions between innovation, ...

FDA and HHS officials have publicly attacked Uniqure, the biotech company seeking approval for a Huntington’s disease treatment, and accused it of lying about requests made by the FDA for additional ...

UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior ...

Subjecting participants to invasive procedures without the prospect of therapeutic benefit is unjustifiable,” says the CEO of ...