Children with Angelman syndrome respond to different types of rewards to promote learning, and the preferred type may vary based on the genetic cause, a new study indicates. The study, “Refining the Behavioral Phenotype of Angelman Syndrome: Examining Differences in Motivation for Social Contact Between Genetic Subgroups,”…
News
Beginning in July, a global 24-hour emergency and urgent care hotline will be available for provider-to-provider consultations to appropriately manage issues, particularly seizures, related to Angelman syndrome. The free hotline is funded through an Emergency Care Consortium grant provided by the Foundation for Angelman Syndrome Therapeutics (FAST). The…
Two U.S.-based Angelman syndrome (AS) nonprofits have created a working group that aims to further awareness and treatment of the rare genetic disorder through legislative advocacy. The Foundation for Angelman Syndrome Therapeutics, known as FAST, and the Angelman Syndrome Foundation, called ASF, have formed a legislative group…
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…
Ionis Pharmaceuticals intends to launch a clinical trial later this year to test ION582, a potential Angelman syndrome therapy. The Phase 1/2a trial, designed to enroll up to 50 patients, is expected to start in the second half of 2021, Ionis wrote in a letter to the…
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…
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
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…
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