Alzheimer's disease (AD) is a debilitating neurodegenerative disorder that causes progressive memory loss and a decline in mental (i.e., cognitive) abilities. Statistics suggest that between 500,000 ...

Inherited retinal degenerations (IRDs) are a group of genetic disorders that lead to progressive vision loss as the light-sensing cells of the eye—the photoreceptors—die due to mutations in genes ...

uniQure N.V. leverages AAV technology to develop gene therapies for liver and CNS diseases, with one FDA-approved treatment and multiple investigational drugs in its pipeline. Despite promising ...