Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial

Abstract Gaucher disease type 3 is a chronic neuronopathic disorder with wide-ranging effects, including hepatosplenomegaly, anaemia, thrombocytopenia, skeletal disease and diverse neurological manifestations. Biallelic mutations in GBA1 reduce lysosomal acid β-glucosidase activity, and its substrat... View More