Chief Clinical Officer, Dascena
Joseph Buxbaum, Ph.D.
Director, Seaver Autism Center
Professor, Psychiatry, Neuroscience, Genetics and Genomic Sciences
Vice Chair for Research and Vice Chair for Mentoring, Psychiatry, Icahn School of Medicine at Mount Sinai
Heather Mefford, M.D., Ph.D.
Full Member, St. Jude Children’s Research Hospital
David Ledbetter and Joseph Buxbaum discussed whether there are genes for which mutations confer risk specific to autism or whether these genes are really conferring general risk of disrupted brain development. The discussion was moderated by Heather Mefford.
Small molecules, genes and antisense oligonucleotides: Industry perspectives on treatment development for ASD
Vice President, Head of Clinical Science, Axial Therapeutics
Stuart Cobb, Ph.D.
Chief Scientific Officer, Neurogene; Research Fellow, University of Edinburgh
Yael Weiss, M.D., Ph.D.
Vice President, Business Development, Ultragenyx
Randy Carpenter, M.D.
Chief Medical Officer, Rett Syndrome Research Trust; Co-Founder, Allos Pharma
Federico Bolognani, Stuart Cobb, and Yael Weiss joined a panel to discuss new industry developments on the use of small molecules, gene therapy and antisense oligonucleotides as treatment approaches for autism spectrum disorders (ASD). The panel discussion was moderated by Randall Carpenter.
Adjunct Senior Research Fellow, Institute for Molecular Bioscience
Affiliate Professor, Queensland Brain Institute, The University of Queensland
Jake Gratten and Naomi Wray presented findings from the Australian Autism Biobank study, an initiative to establish an Australian resource of biospecimens, phenotypes and genomic data for autism research.
Elizabeth Berry-Kravis discussed refinements in clinical trial design and outcome measures that contributed to the success of a recent phase II trial for fragile X syndrome.
Evan Eichler discussed his research on the genetics of autism and related neurodevelopmental conditions.
Emmanuel Mignot discussed sleep biology as well as sleep disorders and their impact. He presented a link to what is known on the genetics of sleep and sleep disorders. He emphasized the need for large scale objective sleep recording studies with genomic and proteomic analysis to better understand the molecular pathways regulating sleep and circadian biology.
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