This year marked a pivotal moment in the quest to treat Huntington’s disease, a rare but devastating form of dementia. Scientists found that an experimental gene therapy slowed the condition’s ...

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency. The ...

Scientists at the University College London (UCL) have developed a novel therapy that helps treat patients with T cell acute lymphoblastic leukemia (T-ALL). This form of therapy used genome editing ...

Dec 9 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening immune disorder. The therapy, Waskyra, was approved for ...

A sign hangs in front of the world headquarters of Vertex Pharmaceuticals in Boston, Massachusetts, U.S., October 23, 2019. REUTERS/Brian Snyder Dec 6 (Reuters) - Vertex Pharmaceuticals said on ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Ollie Chu was three years old when he received an infusion that would change his life. Born with a rare inherited condition called Hunter syndrome, Ollie’s body couldn’t produce an enzyme that breaks ...

Intramuscular injection of a gene therapy known as AMG0001 (Collategene, AnGes Inc.) significantly decreased healing time and increased healing rates of neuroischemic ulcers compared with placebo in ...

“Bubble boy disease” was once a death sentence. A scientific breakthrough changed that. By Simar Bajaj A common cold was enough to kill Cora Oakley. Born in Morristown, N.J., with virtually no immune ...

The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...