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…
Living with a chronic illness comes with challenges people often expect, such as regular medical testing, monitoring, and the occasional surprise. But one thing I’ve come to appreciate over the years is the value of having the right doctors in your corner. For people with rare conditions like Gaucher…
