Risk of “acute, serious, and life-threatening” heart inflammation is now on the label of Sarepta's gene therapy for Duchenne ...

Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.

Sharonville, Ohio, celebrates the opening of the new Cincinnati Children's Applied Gene and Cell Therapy Center.

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year.

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...

Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed ...

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available.

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

A Greer couple is hosting a fundraiser to support a costly gene editing procedure for their two children, who suffer from a ...

The US FDA has tightened the use of a Duchenne muscular dystrophy gene therapy after two teenage patients died from acute ...

A rare disease that has cut off children from the outside world now potentially has a cure. A clinical trial to treat severe ...