The U.S. Food and Drug Administration (FDA) has granted priority review to an application for venglustat, an experimental substrate reduction therapy (SRT) that Sanofi is developing for Gaucher disease type 3. The FDA grants priority review to applications for therapies that may offer significant improvements for serious…
Sometimes it feels strange to write publicly about Gaucher disease because, most days, I don’t feel particularly sick. That’s not something people usually expect to hear from someone with a rare disease. There’s often an assumption that chronic illness has to feel constant and consuming, that it should always…
I was told many years ago that I had an actual expiration date. Put that way, it makes me think of a banana in the grocery store eventually going bad. I know it sounds a bit dark, but that’s what comes to mind. When I was 18 months old, my…
