Angelman Syndrome is a rare genetic disorder caused by UBE3A gene mutations, leading to developmental delays, speech issues, seizures, and distinct behaviors like frequent laughter and hyperactivity.
Dr. Eric Levine’s cutting-edge autism research at UConn School of Medicine is reprogramming the donated skin or blood cells ...
Dr. Eric Levine’s cutting-edge autism research at UConn is reprogramming the donated skin or blood cells of Angelman or Dup15q syndrome patients to develop them in the lab into brain cells that ...
Just before Immy's third birthday, the family finally received an answer: Angelman syndrome, a rare neurogenetic disorder causing global developmental delays. The diagnosis left them wondering if ...
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 ...
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.
The global Phase 3 Aspire study will enroll approximately 120 children ages 4 to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Participants ...