In a major setback, Uniqure said that the timing of when it can file its Huntington's treatment for FDA approval "is now ...

The FDA’s abrupt shift on UniQure’s treatment reflects an agency that, under current leadership, is as unpredictable as its been in years, some analysts said.

Last month, “historic positive results” from uniQure’s gene therapy snapped the Huntington’s community out of years of failure. As the biotech prepares to submit for FDA approval, BioSpace looks at ...

Huntington’s disease (HD) is a condition that is hereditary and causes ... According to the National Health Service, early signs may include trouble focusing or organizing tasks, forgetfulness, ...

Families are elated by news of the first treatment for Huntington's disease - but when and how will the NHS fund it?

Why do some brain cells die first in HD? New study shows changes in gene activity begin at birth in mice, especially in striatum & cortex—mapped with spatial & single-cell tools. A new study in mouse ...

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 ...

The latest update is out from uniQure ( (QURE)). On November 3, 2025, uniQure announced a regulatory update regarding its investigational gene therapy for Huntington’s disease, AMT-130, following a ...

TAMPA, Fla. (WFLA) — For the first time, scientists have successfully treated someone suffering from Huntington’s disease, a fatal genetic illness with no cure. The Mayo Clinic reports that the ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

MRI of a brain with Huntington's Disease. Every week neurologist Victor Sung sees people with Huntington’s disease, a rare and devastating neurodegenerative disorder, at his clinic at the University ...