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

By Michael Erman and Puyaan Singh March 5 (Reuters) - A senior FDA official called UniQure's experimental treatment for ...

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

uniQure held a Type A meeting with the FDA at the end of January and, in a statement released today, said the agency "cannot agree that data from the Phase I/II studies, compared to an external ...

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new trial to prove the therapy actually helps Huntington’s patients. The ...

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

UCL and uniQure report the first treatment to slow Huntington’s disease with gene therapy AMT-130. High-dose patients had 75% less progression over three years compared with standard care. uniQure ...

Researchers have made an important Huntington's disease discovery. They have found that the protein mutated in patients with Huntington's disease doesn't repair DNA as intended, impacting the ability ...

Huntington’s disease (HD) is one of those rare conditions that affects not just the patient but the entire family—medically, emotionally and genetically. This inherited brain disorder causes gradual ...

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 ...

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