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.
Hiding in Plain Sight: Dystonia in Angelman Syndrome
Explore Dr. Robert Carson’s insights on dystonia in Angelman Syndrome, its prevalence, importance of recognition, treatment options, and the role of video documentation in diagnosis.
GTP-220: A Gene Replacement Therapy Being Advanced for Angelman Syndrome
Explore the progress of GTP-220, a gene replacement therapy for Angelman Syndrome, as discussed by Dr. Jim Wilson. Learn about the challenges, disruptive potential, and future plans for this groundbreaking research.
From Benchside to Bedside: Collaboration Leads to Acceleration for Novel Delivery of CRISPR Technology
Explore the exciting advancements in CRISPR technology delivery, from benchside to bedside. Learn about the collaborative efforts, preclinical studies, and the journey of drug development.