The Novel Large Deletion Mouse Model of AS: How Can it Help us in Drug Development for Angelman Syndrome?
Explore Dr. Xiaona Lu’s research on the novel large deletion mouse model of Angelman Syndrome, its generation using CRISPR genome editing, and its potential in drug development.
Stronger Together: The Importance of Collaboration in Rare Disease Advocacy
Explore the importance of collaboration in rare disease advocacy with Annie Kennedy. Learn about the Every Life Foundation’s work, policy priorities, and tools for advocacy. Together, we can make rare not rare.
Stem Cells in Focus: The Role of Glia Cells in a Potential Treatment for Angelman Syndrome
Explore the role of glial cells in treating Angelman Syndrome with Dr. Alvin Wang. Learn about the use of iPSCs in disease study and promising research funded by FAST.
Progress Toward ATF and CRISPR Therapies for Angelman Syndrome
Explore Dr. Dave Segal’s research on ATF and CRISPR therapies for Angelman Syndrome, highlighting gene-targeted treatments and the potential to restore UBE3A expression.
Patient Focused Drug Development
Explore the importance of patient-focused drug development and advocacy for the rare disease community. Learn how engaging with regulators, Congress, and stakeholders can make a difference in patients’ lives.
Measuring Communication Ability in Clinical Trials: A Caregiver-Centered Approach for Angelman Syndrome
Explore Dr. Bryce Reeve’s development of the Observer-Reported Communication Ability (ORCA) measure for Angelman Syndrome. A caregiver-centered tool for clinical trials, capturing unique communication methods.
It’s a Small World: Why FAST Is Global
Explore the global reach of FAST (Foundation for Angelman Syndrome Therapeutics), its challenges, and ambitions for the coming year. Discover how FAST is making significant progress towards finding a cure for Angelman syndrome.
How FAST’s Laser Focus Benefits Translational Efforts in Neurodevelopmental Disorders More Broadly: The View from a Large Private Research Funder
Explore Dr. John Spiro’s insights on how FAST’s focus benefits neurodevelopmental disorder research, as shared at the 2023 FAST Science Summit. Learn about the Simons Foundation’s role and key breakthroughs in the field.
How Endpoints are Used for Drug Approval
Explore the role of endpoints in drug approval, their importance in clinical trials, and how caregiver input shapes the process. Learn about the ABOM consortium’s efforts in improving treatments for Angelman syndrome.
Home-Based Video Capture to Assess Function in People Living with Angelman Syndrome
Explore the innovative use of home-based video capture to assess function in individuals with Angelman Syndrome. This method offers a sensitive, reliable outcome measure, improving clinical trials outcomes.