For children and adults with Gaucher disease type 1 who are clinically stable on enzyme replacement treatment (ERT), spacing treatment to every three to four weeks appears to work as well as the usual every-two-week schedule, according to a study using data from France. Because spacing treatment also…
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…
