Colin Farrell shared an update on his eldest son James, who has a genetic disorder called Angelman syndrome, in a new interview that aired Monday on "Good Morning America". Farrell said his son's ...
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.
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?
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.
Considering taking supplements to treat angelman syndrome? Below is a list of common natural remedies used to treat or reduce the symptoms of angelman syndrome. Follow the links to read common ...
At the Foundation for Angelman Syndrome Therapeutics (FAST) 17 th Annual Global Science Summit in Orlando, FL, Encoded will present data showing that ETX201 was well tolerated in non-human ...
(RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome. The Phase 1/2 data showed improvements ...