Art for Angelman 2024: Our Biggest Fundraiser of the Year
We are thrilled to share the incredible success of Art for Angelman 2024, which raised a record breaking £66,000 for FAST UK. This outstanding achievement makes it our biggest fundraiser of the year.
miRNA-based UBE3A Unsilencing for a Potential One-Time, AAV-Mediated Gene Therapy for Angelman Syndrome
Explore ENCODED’s innovative AAV-mediated gene therapy for Angelman Syndrome. Harnessing the power of gene regulation, they aim to unlock the potential of gene therapy for pediatric CNS disorders.
Using CRISPR activation (CRISPRa) to Upregulate the Existing Gene Copies as a Novel Therapy for the Deletion Genotype of Angelman Syndrome
Explore Dr. Nadav Ahituv’s innovative approach using CRISPR activation (CRISPRa) as a potential therapy for Angelman Syndrome. Discover the power of gene regulation in treating genetic diseases.
Updates on ALDEBARAN, a Phase 2a Trial in Angelman Syndrome
Get the latest updates on the ALDEBARAN Phase 2a trial studying the effects of Alogabat on Angelman Syndrome deletion patients. Presented by Roche Pharmaceuticals at the 2023 FAST Science Summit.
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.