Brantley was born with MECP-2 Duplication Syndrome. Many Americans first learned about this progressive, neurological disorder in 2018, when NBC’s Richard Engel first announced this his son had been ...
Northwestern Medicine scientists have identified the cause of a genetic subtype of autism and schizophrenia that results in social deficits and seizures in mice and humans. Scientists have discovered ...
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y ...
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