VPRIV (velaglucerase alfa), an enzyme replacement therapy (ERT) for Gaucher disease, can be safely administered by patients or caregivers without posing additional safety risks relative to infusions administered by a healthcare provider, a study showed. ERT self-administration was also safe in people with Fabry disease who received…
A gene therapy designed to simultaneously deliver a healthy version of the GBA1 gene and the GDNF gene to cells in the brain eased neurological problems, prevented nerve cell death, and extended survival in a mouse model of Gaucher disease, a study shows. The animals were designed to…
Treatment with Cerdelga (eliglustat), a substrate reduction therapy approved in the U.S. for adults with type 1 Gaucher disease, was safe and showed therapeutic potential in children with the condition, a real-world study finds. “This case series highlights real-world experience with [Cerdelga] in pediatric [Gaucher type 1] patients,…
