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

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

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

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

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

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

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

The FDA will mandate its strictest safety warning on Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, ...

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

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

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

Krystal Biotech drives Vyjuvek growth, premium margins, global expansion, a $1B goal, and pipeline data ahead with a $219.50 ...