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Researchers at Baylor College of Medicine have linked specific variants or mutations of the gene myocardin-related transcription factor B (MRTFB) with a novel neurodevelopmental disorder.
Sio Gene Therapies, a clinical-stage company focused on developing gene therapies for neurodegenerative diseases, presented positive interim data from its Phase I/II study of AXO-AAV-GM1, an adeno-associated viral vector-based gene therapy candidate for GM1 gangliosidosis that was licensed from UMass Chan.