GTX-102 Phase 1/2 Clinical Trial Update, Development of an ASO for Angelman Syndrome: Science and Regulation
Get the latest on the GTX-102 Phase 1/2 clinical trial for Angelman syndrome treatment. Discover trial protocol, safety concerns, preliminary results, and future directions.
Engineering Human Stem Cell Models for Multiple Angelman Syndrome (Epi)Genotypes
Explore the groundbreaking research at North Carolina State University on engineering human stem cell models for multiple Angelman Syndrome genotypes and epigenotypes. Discover our unique approach and dedication to understanding and addressing this complex condition.
Development of Rare Disease Therapies: Overcoming Challenges
Explore the journey of developing rare disease therapies with Emil Kakkis, founder of Ultragenyx. Learn about the challenges, solutions, and the importance of perseverance in this field.
hUBE3A-AAV9 Gene Replacement Therapy for Angelman Syndrome: Progress Toward the Clinic
Explore the progress in gene therapy for Angelman Syndrome with Dr. James M. Wilson. Discover the potential, development stages, challenges, safety considerations, and promising results of clinical trials.
Update on Progress Around the World from FAST Global
Get the latest updates on FAST Global’s progress in accelerating the mission to cure Angelman Syndrome. Learn about their initiatives, collaborations, and achievements worldwide.
Toward Universal Newborn Screening for Angelman Syndrome: The Early Check Approach
Explore the Early Check program’s efforts in North Carolina to implement newborn screening for Angelman syndrome and other conditions. Learn about its goals, collaborative approach, and impact on public health policy.
The Importance of Patient Input for Coverage and Payment of New Therapies
Explore the importance of patient input in coverage and payment of new therapies with experts Sarah Pitluck and John Jarvis. Learn about pricing strategies, patient advocacy, and the role of surveys in improving access to treatments.
The Development of Rare Disease Therapeutics: Compassion and Transparency
Explore the development of rare disease therapeutics with Dr. Emil Kakkis, CEO of Ultragenyx, emphasizing compassion, transparency, and patient-centered approach in clinical trials.
Roche Angelman Syndrome Program Update – 2022
Get the latest updates on Roche’s Angelman Syndrome program, including the progress of Rugonersen, TANGELO Phase 1 drug study, PET imaging study, and the introduction of Alogabat.
Researchers Panel Discussion and Audience Q&A
Join the 2022 FAST Science Summit’s panel discussion on Angelman syndrome research and treatment. Hear from experts addressing audience questions and sh