Breaking the Mold: How Patient Groups like FAST are Reshaping Drug Development in Rare Disease
Explore how patient groups like FAST are revolutionizing drug development in rare diseases. Discover the importance of prevalence, progress, pathways, and partnerships in this process.
Angelman Syndrome Biomarker and Outcome Measure Consortium: What’s the Hype? Why Does it Matter So Much?
Explore the Angelman Syndrome Biomarker and Outcome Measure Consortium’s efforts to improve lives through the development of biomarkers and outcome measures for this rare neurodevelopmental disorder.
Advancing Human Stem Cell-Derived Platforms for Angelman Syndrome Research
Explore the advancements in Angelman Syndrome research through human stem cell-derived platforms. Discover diverse models, cerebral organoids studies, and high-throughput drug testing methods.
A Family-Centered Approach to Measuring Communication Ability within Clinical Trials
Explore Dr. Christie Zigler’s development of the ORCA measure, a unique tool for evaluating communication ability in clinical trials, specifically for Angelman Syndrome. Learn about its creation, validation, and future plans.
An Update on HALOS Clinical Trial in Individuals Living with Angelman Syndrome
Get the latest update on the HALOS clinical trial for Angelman syndrome treatment. Discover the approach, preliminary findings, and future steps in this groundbreaking research by Ionis Pharmaceuticals.
Pharma Industry Update session
Get the latest updates on Angelman syndrome drug development and clinical trials from the 2022 ASF Family Conference. Learn about progress from Roche, Ultragenyx, PTC Therapeutics, Ionis, Biogen, and Neuren Pharmaceuticals.
Clinical Expert Panel session at the 2022 ASF Family Conference
Join the Clinical Expert Panel at the 2022 ASF Family Conference for insights on managing Angelman syndrome. Learn about common medical issue
Behavior and Anxiety in AS
Explore the common behavioral issues and anxiety in individuals with Angelman Syndrome. Learn about their frequency, severity, causes, and effective strategies for management and intervention.