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3 results for "syngap1"
A groundbreaking treatment for SYNGAP1, a rare genetic disease causing seizures and developmental delays, has entered human trials for the first time. The therapy aims to restore a crucial brain protein that 1,800 identified patients worldwide currently lack.
Patient groups from 10 countries just joined forces to speed up treatments for SYNGAP1, a rare genetic disorder affecting children worldwide. The new collective will coordinate research funding, clinical trials, and treatment access across continents.
A PhD student at MIT is developing a breakthrough gene therapy for children with SYNGAP1 disorder, a rare genetic condition that causes seizures starting as young as 4 months old. Early tests in mice have eliminated seizures completely.