Study: "Profound" diagnoses of autism are far outnumbered by milder cases.

Published Date: 26 Apr 2023

Health officials have wondered how many U.S. children are being diagnosed with autism as it becomes more common. S. How many children experience more severe symptoms like a very low IQ and verbal immaturity, and how many kids have relatively mild symptoms?

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The phase 3 study aims to confirm findings from the HEALEY ALS platform trial in patients with early-stage rapidly progressive ALS, which suggested potential benefits of pridopidine across multiple domains.

New data from the open-label extension of Harmony Biosciences’ Phase 3 ARGUS trial show that EPX-100 (clemizole) achieved a median 50% reduction in countable motor seizures in patients with Dravet Syndrome.

Capricor Therapeutics anticipates that detailed phase 3 data from the HOPE-3 study will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.

Michael Sperling, MD, Baldwin Keyes Professor of Neurology at Vice Chair for Research at the Sidney Kimmel Medical College, spoke about the significance of receiving the Founders Award and the current state of epilepsy.

Long-term treatment with z-rostudirsen over 24 months showed sustained functional improvements across all end points in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

TRN-257, a treatment in development for adults with narcolepsy and idiopathic hypersomnia, is designed to reduce daily sodium exposure relative to existing higher-sodium therapies.

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