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.
An Update on HALOS Clinical Trial in Individuals with Angelman Syndrome
Explore the latest updates on the HALOS clinical trial for Angelman syndrome by Ionis Pharmaceuticals. Learn about the development of ION582 and its potential to treat this condition.
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.
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.