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

12:06 PM ETDeveloping

Health Desk

In Short: The failure of an experimental drug for Angelman syndrome, a rare genetic condition causing severe developmental delays, has been a significant setback for patients and the drug’s maker, Ultragenyx, which announced the

5-year-old Mexican girl with Angelman syndrome (cropped) — Photo: Yokoyama-Rebollar E, Ruiz-Herrera A, Lieberman-Hernández E, Del Castillo-Ruiz V, Sánchez-Sandoval S, Ávila-Flores SM, Castrillo JL - Mol Cytogenet (2015) / Wikimedia Commons

The failure of an experimental drug for Angelman syndrome, a rare genetic condition causing severe developmental delays, has been a significant setback for patients and the drug’s maker, Ultragenyx, which announced the outcome of its late-stage trial earlier this week.

Angelman syndrome is a rare genetic disorder that affects about 1 in 15,000 people, characterized by developmental delays, severe speech impairment, and distinctive behavioral traits.

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“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.

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