News

No safety concerns seen with VPRIV self-administration: Study

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…

Cerdelga shows benefits in children with Gaucher disease type 1: Study

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,…

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

FDA Grants Orphan Drug Status to M6P Gene Therapy

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to two gene therapies that M6P Therapeutics is developing — one intended for Gaucher disease, the other aiming to treat the inherited metabolic disorder mucolipidosis. The regulatory agency also awarded six rare pediatric disease designations to…