There are days when Gaucher disease feels like an interruption more than anything else. Not a crisis, and not even a central focus — just something that gets in the way. Life, on the other hand, is very full. I’m a stay-at-home mom to an active toddler while also…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy status to Sanofi‘s experimental oral medication for Gaucher disease type 3, venglustat, which has shown potential in a clinical trial for improving measures of neurological function in people with this form of the genetic disorder. While there…
Treatment with VPRIV (velaglucerase alfa) was generally safe and showed benefits — including stable blood levels — among children and adolescents with Gaucher disease type 1, a new review of published studies has found. Evidence also suggests the approved enzyme replacement therapy (ERT) may help manage nonneurological…
