Gene-Based Therapy Restores Cellular Development and Function in Brain Cells From People With Timothy Syndrome

Gene-Based Therapy Restores Cellular Development and Function in Brain Cells From People With Timothy Syndrome

NIH-supported study shows potential treatment pathway for neurodevelopmental disorder April 24, 2024 • Press Release In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome , an... Read more »
Researchers Expand Understanding of Genetic Mechanisms Underlying Fragile X Syndrome

Researchers Expand Understanding of Genetic Mechanisms Underlying Fragile X Syndrome

January 18, 2024 • Research Highlight Fragile X syndrome  is a genetic disorder caused by the gene FMR1. It is the most common form of inherited intellectual disability and often co-occurs with... Read more »
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