Angelman syndrome is a rare disease that disrupts the normal development and function of cells, particularly in the nervous ...
Colin Farrell opened up about the mission of The Colin Farrell Foundation and caring for his son James, who has Angelman ...
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.
Shortly thereafter, diagnoses of Angelman syndrome began to rise. Today, the disorder is known to occur in approximately one in every 15,000 to 20,000 people, and it has been reported all over the ...
"We have to help people understand how dire the situation is for so many," Farrell, who serves as the foundation's president, said during a speech at the group's Dec. 7 gala. Angelman syndrome ...
All told, 97% of people in the medium and high dose groups saw an improvement in overall symptoms measured using the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C ...
Overall, 97% of people in the medium and high dose groups assessed in the study saw an improvement in overall AS symptoms as measured by the Angelman Syndrome Clinical Global Improvement Change ...
(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 ...