2017 FAST Science Summit

• AAV-mediated gene therapy approach: Agilis Biotherapeutics
  Explore Agilis Biotherapeutics’ AAV-mediated gene therapy for Angelman Syndrome. Learn about their research, preclinical studies, and the challenges in bringing this innovative treatment to clinical trials.
• Approaching the clinic: nutritional support for patients with Angelman Syndrome
  Explore Disruptive Nutrition’s clinical trial on a ketone ester supplement for children with Angelman Syndrome. Learn about its potential to improve seizure control and overall quality of life.
• Gene therapy round table: ask the experts
  Join our Gene Therapy Round Table where experts discuss strategies, challenges, and potential treatments for Angelman Syndrome. Your involvement is crucial for advancing research in this field.
• In the clinic: Ovid Therapeutics-OV101 and the engagement with the Angelman community
  Explore Ovid Therapeutics’ commitment to Angelman Syndrome, their innovative OV101 program, and their active engagement with the Angelman community. Learn about their ongoing STARS trial and future plans.
• Keynote: AAV-mediated gene therapy to the central nervous system: prospect for Angelman syndrome
  Explore Dr. Jim Wilson’s insights on AAV-mediated gene therapy for rare diseases like Angelman syndrome. Learn about its potential, challenges, successes, and the importance of continued research.
• Modifying the Bayley test to benefit clinical trials in Angelman syndrome
  Explore the work of Anjali Sadhwani and Dr. Anne Wheeler on modifying the Bayley test for assessing children with Angelman Syndrome. Learn about their initiatives to improve the test’s effectiveness.
• Overview of the FAST research agenda for gene therapy and genetic editing in Angelman syndrome
  Explore the FAST research agenda for gene therapy and genetic editing in Angelman syndrome. Understand the therapeutic approaches, progress made, and how to support research efforts.
• The Angelman Biomarkers and Outcome Measures Alliance (A-BOM)
  Explore the Angelman Biomarkers and Outcome Measures Alliance’s work in developing outcome measures for Angelman syndrome. Learn about their research, clinical trials, and how to get involved.
• The Global Angelman Syndrome Registry – 2017
  Explore the Global Angelman Syndrome Registry, a crucial tool for data collection and research advancement. Learn about its purpose, participation process, and how it’s improving outcomes for individuals with Angelman Syndrome.
• Therapeutic development and outcome evaluation using a disease concept model in AS
  Explore Roche’s innovative approach to developing transformative therapies for Angelman Syndrome, focusing on biomarkers, outcome measures, and a disease concept model.