NewsDialy
A late-stage trial for an experimental Angelman syndrome drug came back negative this week, dealing a major blow to patients and to the company behind it.
Late-stage, in clinical testing, means the final round of human trials a medicine must complete before the maker can ask regulators to approve it for public use.
Ultragenyx, which developed the drug, announced the outcome. Angelman syndrome is a rare genetic disease.
It causes severe developmental delays, and families living with it have had few treatment options available to them. The trial that just failed was testing whether a genetic medicine could change that.
Keep reading