Tag Archives: After

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 »

NICE blames BioMarin for Brineura rejection after price talks break down

BioMarin and England’s cost-effectiveness gatekeeper have been negotiating for a year, but that hasn’t helped the rare-disease specialist secure reimbursement for its Batten disease drug Brineura. Friday, the National Institute for Health and Care Excellence (NICE) published draft guidance sticking with its earlier decision not to endorse Brineura in children with the disease, an inherited… Read More »