Author Archives: Patricia Inacio PhD

Study Links Missing Gene to Severe Seizures in Angelman Children

Epileptic seizures occur earlier and more frequently, leading to more hospitalizations, in children with Angelman syndrome due to a missing UBE3A gene, according to a retrospective analysis of a natural history study. The study, “Clinical Characterization of Epilepsy in Children With Angelman Syndrome,” was published in the journal…

Trial for GTX-102 to Open Enrollment Soon in the UK

The United Kingdom’s Medicines Healthcare Products Regulatory Agency (MHRA) has authorized a Phase 1/2 clinical trial to test the investigational therapy GTX-102 in children and adolescents with Angelman syndrome. The open-label KIK-AS trial (NCT04259281), which also has been given a green light in Canada, is expected to open enrollment in the…

KIK-AS Phase 1/2 Trial of GTX-102 Can Resume Enrollment in Canada

Health Canada has given the green light for a Phase 1/2 clinical trial to resume enrollment to test the investigational therapy GTX-102 in children and adolescents with Angelman syndrome in Canada. This follows an amendment to the trial’s protocol, submitted by GeneTx Biotherapeutics and Ultragenyx Pharmaceutical, the therapy co-developers, after…

Cannabinoid BIO017 Receives FDA’s Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BIO017, a candidate therapy for Angelman syndrome being developed by Biom Therapeutics, the company has announced. Orphan drug status is designed to encourage therapies for rare and serious diseases, through benefits such as seven…