For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman ...

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.

"Angelman Syndrome is a neurogenetic disorder," said Weston's mother Felicia Warner. "It affects a lot of different things. It affects their balance, a very high percentage has seizures ...