Patricia Inacio, PhD,  science writer—

The first patient has been dosed in Canada as part of a Phase 1/2 clinical trial of the investigational therapy GTX-102 in children and adolescents with Angelman syndrome. The open-label KIK-AS trial (NCT04259281), which is also underway in the U.S. and U.K., has enrolled three other patients who are scheduled to…

A professor at the University of California (UC) Davis Health has been awarded a $1.36 million Discovery Stage Research Projects (DISC2) grant to develop a gene therapy for Angelman syndrome, according to a university press release. David Segal, PhD, a professor in the departments of biochemistry and molecular…

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…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

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…

Ovid Therapeutics is stopping its clinical program for OV101 (gaboxadol), a candidate therapy for both Angelman and fragile X syndrome, in favor of focusing its resources on OV882, a potential RNA therapy for Angelman patients, it announced in a corporate update. The…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

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…

The Foundation for Angelman Syndrome Therapeutics (FAST) has awarded researchers with a grant to develop novel methods to accurately assess gait and mobility across age groups in patients with Angelman syndrome. The research project is led by Jill L. Silverman, PhD, at the University of California, Davis, and Jessica…