Science Summit
Becky Burdine, Ph.D. ~Introduction/Therapeutics in Angelman Syndrome
Ovid Therapeutics ~The use of Gaboxadol (OV101) in the treatment of Angelman Syndrome. Down stream therapeutics working at the neuronal synapse. Discussion of future clinical trials.
Agilis Biotherapeutics ~ The use of gene therapy for the production of UBE3A in the treatment of Angelman Syndrome. Discussion of future clinical trials.
Disruptive Nutrition ~ The use of Ketone Supplements for the nutritional management of Angelman Syndrome. Discussion of pre-clinical trial outcomes and future human clinical trials.
Vivint ~ The use of a smart technology home security system to protect your loved ones.
FAST FIRE Team
The most aggressive, novel, innovative research initiative whose goal is to identify treatments for Angelman Syndrome.
- Edwin Weeber, Ph.D. – Behavioral differences seen in AS, new potential outcome measures in AS and synaptic function in the AS model.
- Scott Dindot, Ph.D. – AS drug screening, mechanisms regulating imprinting of UBE3A and new animal models of AS.
- Anne Anderson, MD – Seizure and behavioral phenotypes in the AS mice and how this information will be used in preclinical trials.
- David Segal, Ph.D. – Progress in designing epigenetic regulators for persistent UBE3A activation.
- Kevin Nash, Ph.D. – Protein replacement therapy using gene delivery.
Educational Summit
Linda Burkhart & Gayle Porter ~
Information to clarify desirable long-term outcomes and evidence informed practices towards the development of accessible, autonomous communication for children who use AAC.
David Koppenhaver & Susan Norwell ~
Key principles and strategies including structured shared reading, predictable chart writing, independent emergent reading and writing, and early skills instruction. The use of Core Vocabulary and Soft-Tech Reading Page Sets and how they support communication during shared reading. Decision tree for determining when emergent literacy or conventional literacy instruction is most appropriate.
Q&A/Panel Discussion