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 ...

Two complementary studies led by researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS ...

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 ...

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

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

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.

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

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

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