The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient populations.

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 to successfully use a gene therapy technique to insert a ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial results. The drug, called zorevunersen, holds promise, especially for people with the condition who don’t respond to existing treatments such as antiseizure medications.

Many psychiatric disorders are more biologically related than previously understood, having more genetic similarities than differences, a new study shows. More research is needed to understand how these genetic similarities manifest in different groups of ...

If you haven’t heard of the genetic disorder Heterotaxy, a new children’s book released by a Connecticut teacher and her cousin is spreading awareness. Connecticut

Nick Jonas' neighbour Maya Kibbel recently died of Wilson's disease at the age of 30. The disease is a rare genetic disorder where copper gets accumulated in the body, affecting different organs.

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 allows for the early identification of hereditary ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way for the next generation of gene editing medicines. The approach, which is described in a Nature paper published March 11,