2018 FAST Science Summit

• Angelman Overview
  Explore Angelman Syndrome’s genetics and therapeutic options with Allyson Berent, a mother and chief science officer of FAST. Understand the disorder and promising treatments.
• Assessing Communication Ability of Children with Angelman Syndrome
  Explore Dr. Bryce Reeve’s research at Duke University on assessing communication abilities in children with Angelman Syndrome, aiming to develop a parent-reported measure for clinical trials.
• Blood Stem Cells: New Gene Therapy Approach for Angelman Syndrome
  Explore Dr. Joe Anderson’s innovative gene therapy approach for Angelman Syndrome using blood stem cells. Discover how this unique strategy could pave the way for future treatments.
• CRISPR – Pros and Cons, Promise, Possibilities, and Concerns
  Explore the potential of CRISPR-Cas in treating genetic diseases with Dr. David Segal. Understand its revolutionary role in gene editing, its therapeutic applications, and the ethical concerns it raises.
• Characteristics of the Ube3a Large Deletion Rat (Legend-Rat)
  Explore the characteristics of the Ube3a Large Deletion Rat, a model for Angelman Syndrome research. Learn about synaptic function, behavior, EEG, and epilepsy from leading experts.
• Gene Therapy for Angelman Syndrome
  Explore Dr. Jim Wilson’s insights on the potential of gene therapy for treating Angelman Syndrome, a rare neurogenetic disorder. Learn about the challenges, progress, and future of gene therapy.
• GeneTx Biotherapeutics. A Novel Approach to Drug Development – The Why, The What, and The How?
  Explore GeneTx Biotherapeutics’ innovative approach to drug development for Angelman Syndrome. Learn about their team, motivation, antisense oligonucleotide drug, and their journey towards an IND application.
• Genotype Matters
  Explore the significance of genotype in Angelman Syndrome with Dr. Art Beaudet. Learn about genetic variations, their impact on symptoms, and potential treatments.
• Keynote Speaker, Dr. Timothy Yu
  Explore Dr. Timothy Yu’s keynote speech at the 2018 FAST Science Summit, highlighting his groundbreaking work in personalized medicine for rare neurodevelopmental disorders like Batten and Angelman Syndrome.
• My Journey Through Drug Development for More Meaningful Change
  Explore Dr. Lauren Black’s journey in drug development for rare diseases like Angelman Syndrome. Learn about FDA regulations, collaboration, and the importance of patient safety in the process.
• New Treatment for AS – IGF-2 Receptor Ligand Reverses Multiple Deficits
  Explore Dr. Alberini’s groundbreaking research on IGF-2 as a potential treatment for Angelman Syndrome. Discover how this memory enhancer can reverse cognitive and motor deficits.
• Nutritional Approaches for Angelman Syndrome: Clinical Trial Update
  Explore the latest clinical trial on ketone supplements for Angelman Syndrome treatment. Learn about the trial’s design, outcomes, and how it aims to improve patient nutrition and metabolism.
• OV101 Development Update: STARS Results and What’s Next
  OV101 Development Update: Positive Results from STARS Study and Next Steps
• PTC Therapeutics – Gene Therapy for Angelman Syndrome
  Explore PTC Therapeutics’ advancements in gene therapy for Angelman Syndrome. Learn about their progress, challenges, and commitment to developing effective treatments for this rare genetic disorder.
• Roche Pharma Research and Early Development
  Explore Roche Pharma’s research and early development in Angelman syndrome. Discover their scientific approach, community collaboration, and plans for endpoint development in rare disease treatment.
• Round Table Panel on the Treatment of Angelman Syndrome
  Explore insights from the 2018 FAST Science Summit’s Round Table Panel on Angelman Syndrome treatment. Learn about potential cures, clinical trials, and future treatment accessibility from leading experts.
• The Global Angelman Syndrome Registry – 2019
  Explore the Global Angelman Syndrome Registry, a crucial project for advancing research on Angelman Syndrome. Learn about its goals, benefits of participation, and how you can contribute.