Avigbagene parvec, a one-time gene therapy being developed by Spur Therapeutics for Gaucher disease type 1, helped four of six adults in a Phase 1/2 study remain off their standard treatment for up to about two years, trial data show. While stopping treatment typically leads to a rapid…
When you’re newly diagnosed with Gaucher disease, you’re given a lot of information. Much of it is well intentioned and medically sound, such as explanations of enzyme levels, treatment options, and what to expect next. What’s explained less often is how much you’ll learn about living with Gaucher along…
Sanofi‘s experimental oral therapy venglustat was better than its enzyme replacement therapy (ERT) Cerezyme (imiglucerase) at easing neurological symptoms in people with Gaucher disease type 3. That’s according to new data from the LEAP2MONO Phase 3 clinical trial (NCT05222906), presented as late-breaking research last week…
