From Genes to Treatment: Using Rare Genetic Disorders to Develop Novel Therapeutics

At the Seaver Autism Center for Research and Treatment’s 21st Annual Advances in Autism Conference, Dr. Kolevzon discussed how gene discovery can lead to modeling systems that illuminate the pathophysiology of autism, which will lead to drug development and novel therapeutics. He emphasized the need for treatment measures specific to the syndrome/phenotype, the clinical utility of biomarkers, and the importance of focusing on functional improvements such as motor skills and language in addition to irritability, aggression, etc. He also suggests using younger subjects, as clinical trials typically start at age five and the brain is more plastic at younger ages. Finally, he recommends longer duration of clinical trials, as he believes the standard 12 weeks may not be long enough to see changes in behavior.

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Alexander Kolevzon, MD.

Alexander Kolevzon, MD

Certifications Child & Adolescent Psychiatry Psychiatry, American Board of Psychiatry and Neurology Clinical Focus Anxiety Attention Deficit Hyperactivity Disorder And Attention Deficit Disorder Autism Education MD ...

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