STAT+: After failure of Angelman syndrome drug, experts see hope in other experimental therapies

The failure of an experimental drug for Angelman syndrome, a rare genetic disease that causes severe developmental delays, was a major blow to patients and the drug’s maker, Ultragenyx, which announced the outcome of its late-stage trial earlier this week. But experts cautioned that there is still reason to think similar experimental medicines might yet succeed. That could potentially pave the way for genetic medicines in other neurological conditions that could restore cognition, communication, and other skills in patients with intellectual disabilities. “I don’t think this really says anything about the other trials that are ongoing,” said Mark Zylka, an Angelman researcher at the University of North Carolina. “I wouldn’t say that just because this one trial fails that means [this is a bad mechanism].” STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe