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

In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed fingers and toes.
Source: National Institute of Mental Health - Category: Psychiatry Authors: Source Type: news