News

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…

FAST, SFARI Partner to Launch International Research Council

The Foundation for Angelman Syndrome Therapeutics, known as FAST, is partnering with the Simons Foundation Autism Research Initiative (SFARI) to launch the International Angelman Syndrome Research Council — or INSYNC-AS — with a goal to “build community and leverage the combined skills of world leaders in neuroscience, clinical…

ORCA, Reliable Measure of Communication for Angelman, in Early Use

A reliable tool for parents or caregivers to judge the quality of nonverbal communication in Angelman syndrome patients is now available, according to the Foundation for Angelman Syndrome Therapeutics (FAST), which supported work that lead to its development. Called Observer-Reported Communication Ability (ORCA), the tool grew from a partnership between FAST and…

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…