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With a nudge from AI, ketamine emerges as potential rare disease drug

Families of kids with a rare disease have waited years for a treatment. In seconds, an AI tool pinpointed an unexpected target: ketamine. ....

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Teen is on front lines of autism clinical trial

Teen is on front lines of autism clinical trial
HACKENSACK, N.J. She lives in a world no one else can enter, unable to speak or interact with others. But 16-year-old Rebecca Singer may be playing an important role in science.
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Rebecca Singer, 15, of Tenafly, New Jersey has a rare genetic disease that causes severe disabilities and autistic symptoms. Here, Singer hesitates a moment as she gets off a school bus while her father Jon Singer greets her at their home. (Mitsu Yasukawa/The Record/MCT)
HACKENSACK, N.J. She lives in a world no one else can enter, unable to speak or interact with others. But 16-year-old Rebecca Singer may be playing an important role in science. ....

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ORYZON Reports Results and Corporate Update for Quarter Ended March 31, 2021

ORYZON Reports Results and Corporate Update for Quarter Ended March 31, 2021
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Mount Sinai and Rumi Scientific team up to advance autism drug discovery

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The Seaver Autism Center for Research and Treatment at the Icahn School of Medicine at Mount Sinai and Rumi Scientific announced today that they will team up to initiate a drug discovery pipeline for rare genetic disorders that carry a high risk of autism. The project will initially involve the development and use of a platform that mimics the development of brain tissues, at high speed, allowing researchers to gain insights into how cells respond to three known genes implicated in autism.
Over the past several years, more than 100 genes have been identified that confer very high risk to autism when mutated, and the Seaver Center at Mount Sinai is one of the lead sites for these autism gene discoveries, said Joseph D. Buxbaum, PhD, Director of the Seaver Autism Center for Research and Treatment at Mount Sinai and Professor of Psychiatry, Neuroscience, and Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai. We are eager to work together ....

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