Gaucher disease may alter how macrophages, key immune cells involved in the disease, produce energy and process fats, according to a new study using computational modeling. Based on their model, researchers identified a few genes that may help regulate these metabolic changes in Gaucher cells. The scientists called for…
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…
