In case you missed it, due to the snowstorm, the Hotel Concord was lit up in blue Saturday to honor International Angelman ...

International Angelman Day is recognized on February 15. The date reflects the genetic defect Angelman Syndrome causes in the 15th chromosome and nods to Feburary’s designation as Rare Disease Month.

Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...

Every morning, eight-year-old Isabelle watches from the window as her two sisters Madison and Olivia leave to walk to school. “Izzy gets so excited, because she thinks she’s going to go. But then, she ...

Dr. Eric Levine’s cutting-edge autism research at UConn School of Medicine is reprogramming the donated skin or blood cells ...

Encoded Therapeutics is shrinking its headcount by 29% as the gene therapy biotech draws resources away from its early-stage ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

to treat Angelman syndrome (AS). "This program has consistently delivered remarkable animal data, reinforcing its potential as a groundbreaking treatment," said Dr. Donald Kohn, Distinguished ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

The data to date, funded by TransformaTx Biotherapeutics, a biotechnology company founded by the Foundation for Angelman Syndrome Therapeutics (FAST), has demonstrated complete symptomatic ...