Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report

Gaucher disease is one of the most common lipid-storage disorders, affecting approximately 1 in 75,000 births. Enzyme replacement therapy with recombinant glucocerebrosidase is currently considered the first-l...

http://jmedicalcasereports.biomedcentral.com/articles/10.1186/s13256-016-1060-y