Type 3 Gaucher disease
Although some individuals with type 3 Gaucher disease may have a very aggressive and rapid downhill course, that is by no means an inevitable outcome. We know of many patients with type 3 GD who mature normally, have normal intellectual acuity, are successful students and workers and, provided that the non-neurological manifestations of the disease are treated properly, can live many years as independent, productive and respected members of their communities.
Abnormal blood counts while on treatment
The current approved treatments for type 1 Gaucher disease are generally so successful that, should patients who have responded well and are continually in compliance with their treatment regimen become anemic, have an unexpected decrease in platelets or a change in their white blood cell, it is most likely something other than Gaucher disease that is causing the problem. Make sure your physician investigates all the possibilities.
Importance of continued research
With current treatments, most people with type 1 Gaucher disease who pursue a healthy, active lifestyle can expect to achieve a normal life expectancy. Nevertheless, it is important to continue to investigate and test new treatment options to adequately address some relatively uncommon late-onset complications such as Parkinsonism and to improve the convenience and lifetime cost of therapy. Ask your physician about ongoing and new clinical trials.