Findings from the LEAP2MONO study support the regulatory submission of venglustat for treatment of type 3 Gaucher disease.
Among previously untreated adults with Gaucher disease type 1, a genetic disease in which there is improper metabolism due to a defect in an enzyme, treatment with the drug eliglustat resulted in ...
Spleen stiffness measurement (SSM) demonstrated strong predictive accuracy for liver decompensation in patients with compensated advanced chronic liver disease (cACLD), with an optimal cutoff of 50 ...