With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Despite modern high-throughput sequencing, the genetic cause of most rare movement disorders remains unclear. A research team ...

Discover how CRISPR genome editing is revolutionizing medicine. Learn the science of Cas9, current clinical trials, and the future of gene editing.

Most of the genetic risk for developing a substance use disorder comes from genes that broadly affect how our brains process ...

Several children in the UAE, facing developmental delays and muscle weakness underwent standard genetic tests only for the ...

A Marion woman is celebrating a victory for those suffering from rare diseases across the state after securing a proclamation from Governor Andy Beshear declaring December 2025 as Brooke-Spiegler Synd ...

In an exciting scientific first, researchers at the Allen Institute successfully designed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders (SRD) in mice. These are a ...

Genetic testing can help doctors diagnose some neurological disorders, such as Huntington’s disease. It can also provide insights into a person’s risk of future health conditions. Genetic testing ...

An immune-related gene has now been tied to a rare inherited neurological disorder, revealing an overlooked pathway in the nervous system.

Researchers analyze 2.2 million genomes to show that addiction risk is primarily driven by broad genes affecting brain wiring and impulse control, not drug-specific traits.