presented results of both in vitro and in vivo studies at the November 2024 FAST Global Summit on Angelman syndrome. The team will be using the new CIRM grant to prepare a pre-IND package ...

"So FAST (Foundation for Angelman Syndrome Therapeutics) and the Angelman Association in Australia worked really hard on our ...

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.

We each get two copies of every gene - one copy from each of our parents. But what happens when one of these genes has been "turned off", or imprinted, and the remaining gene is defective?

Angelman syndrome is caused by a large deletion of the region of the maternal chromosome that contains UBE3A, or by a DNA sequence change (mutation) in the UBE3A gene inherited from the mother.