Tag Archives: gene

Novartis slaps $2M-plus pricetag on newly approved gene therapy Zolgensma—and cost watchdogs approve

Industry watchers have been speculating for months about just how high Novartis would price gene therapy Zolgensma on approval—and just how payers would cover it. And with the FDA’s green light, they now have an answer. The Swiss drugmaker will roll out the drug, a one-time treatment for pediatric patients less than 2 years of… Read More »

Three years after undergoing gene therapy, his ‘last shot’ hit the target

Brenden was the first patient enrolled in what became a nine-patient, four-center trial that also involved patients at the National Institutes of Health (NIH) in Maryland, the University of California at Los Angeles, and Great Ormond Street Hospital for Children in London. The trial, funded by the NIH and the California Institute for Regenerative Medicine,… Read More »

Gene therapy eases Parkinson’s symptoms by rewiring parts of the brain

A treatment for Parkinson’s rewires parts of the brainALFRED PASIEKA/SCIENCE PHOTO LIBRARY By Alice Klein A gene therapy treatment for Parkinson’s disease appears to relieve symptoms by rewiring the brain circuits involved in movement. People with Parkinson’s disease have tremors and muscle stiffness that are caused by overstimulation of a brain area called the subthalamic… Read More »