Angelman Drug Trial Fails, Similar Therapies Still Hold Promise — SkimNews
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Ultragenyx announced earlier this week that its late-stage trial for an experimental Angelman syndrome drug failed, a major blow to both patients and the company.
- Angelman syndrome is a rare genetic disease that causes severe developmental delays in those affected.
- Mark Zylka, an Angelman researcher at the University of North Carolina, said the failure says nothing about other ongoing trials and wouldn't be used to condemn the mechanism.
- Experts cautioned that similar experimental medicines might yet succeed despite Ultragenyx's setback.
- Success in this space could pave the way for genetic medicines in other neurological conditions aimed at restoring cognition, communication, and other skills in patients with intellectual disabilities.
Why it matters: The failure of Ultragenyx's late-stage Angelman trial doesn't invalidate the broader mechanism behind similar therapies, researchers argue. Each experimental treatment must be judged on its own merits, leaving other Angelman drugs and genetic medicines targeting intellectual disabilities still in play for patients and families.
Ask SkimNews
