date:May 04, 2012
o the small number of affected patients, the efficacy of Elelyso was evaluated in a total of 56 patients with Type 1 Gaucher disease enrolled in two clinical trials. Many of these patients continued treatment on a longer-term extension study.
In one multi-center, double-blind, parallel-dose trial, the efficacy of Elelyso for use as an initial therapy was evaluated in 31 adult patients who had not previously received enzyme replacement therapy. Patients were randomly selected to receive Elelyso