While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

In a small trial, a gene therapy injected into the brain slowed the disease by 75 percent over three years. Huntington’s disease is extremely cruel. Symptoms start with random, uncontrollable twitches ...

For the first time, scientists have successfully treated someone suffering from Huntington's disease, a fatal genetic illness ...

Add Yahoo as a preferred source to see more of our stories on Google. An exceptionally rare and fatal genetic disease may finally have a solution. Huntington’s Disease affects one in ten to twenty ...

There is no known cure for Huntington's disease. A genetic mutation creates harmful proteins that accumulate and cause the disease's typical symptoms. A team from the Department of Human Genetics at ...

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

NEW YORK, March 4 (Reuters) - UniQure's trial design for its gene therapy for Huntington's disease did not offer sufficient proof that the drug slowed disease progression, a top U.S. Food and Drug ...

Will Forte, who played MacGruber on Saturday Night Live, and his brother-in-law Doug Modling, who has Huntington's Disease, ...

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...

Amid rising debate within the medical community, the US FDA has spoken out to defend how it reviews and authorizes new ...

AUSTIN (KXAN) — An exceptionally rare and fatal genetic disease may finally have a solution. Huntington’s Disease affects one in ten to twenty thousand Americans. The disease is a mixture of dementia ...