DATA GRAPHICS | Product Development
Gaucher disease pipeline: Data Byte
Three clinical and 11 preclinical candidates move beyond traditional enzyme replacement, aim for wider range of patients
September 30, 2021 12:57 AM UTC
Behind the five marketed therapies for Gaucher disease, all of which treat Type I Gaucher and were approved before 2015, is a pipeline of at least three clinical and 11 preclinical candidates, according to BioCentury’s BCIQ database. Seven of the programs could offer patients a one-time treatment option.
Gaucher disease is a rare genetic disorder that results from an enzyme deficiency that leads to lipid accumulation in liver, spleen, bone marrow and nervous system. ...