Update on the GTX-102 program for Angelman Syndrome
Get the latest update on Ultragenyx’s GTX-102 program for Angelman Syndrome. Learn about the Phase 2 trial, significant improvements observed, and future plans for this promising investigational drug.
Understanding Critical Clinical Outcome Assessments (COAs) Used in Clinical Trials
Explore Dr. Sadhwani’s insights on critical clinical outcome assessments (COAs) in clinical trials, their types, characteristics, and their role in evaluating treatment effectiveness.
Transformatx Update: Hematopoietic Stem Cell Gene Therapy Program
Explore Transformatx’s lentiviral gene therapy program, ube-cel, aimed at treating Angelman syndrome. Learn about its administration process, previous successes, challenges, and future plans.
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.