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

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

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.

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

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

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

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 ...