The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...

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.

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

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

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

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

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

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

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

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

The FDA has added a boxed warning to Sarepta’s Duchenne gene therapy Elevidys after two teens died from liver failure and has ...