Today, the company announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its novel adeno-associated virus (AAV) gene therapy ...

One finisher, Andrew Patterson, thought he would not walk again after he was diagnosed in 2013 with hereditary spastic paraplegia, a disorder that causes weakness and stiffness in the leg muscles.

Robert Wickens was one of motorsport's rising stars when his life was permanently altered in a crash that paralyzed him from the chest down in 2018. Ever since, Wickens has said that his goal is ...

The degenerative brain disease has no cure, and it’s only natural to ask yourself: Is Alzheimer’s hereditary? Meet the experts: James Noble, M.D., an associate professor of neurology at ...

Hereditary angioedema (HAE) is a rare condition that causes fluid to build up throughout your body, triggering sudden and repeated serious swelling. This is the most common type and happens ...

Type 2 diabetes can be inherited, but environmental factors also play a role. Not everyone with a family history of type 2 diabetes will get it, but you’re more likely to develop it if a parent ...