Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1Pramod K. Mistry, Michel Peterschmitt, Atul Mehta et al.|JAMA|2015Cited by 156
Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): Literature review and report of new casesDavid Cassiman, Gerald F. Cox, Manuel Schiff et al.|Molecular Genetics and Metabolism|2016Cited by 122
Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with <scp>G</scp>aucher disease type 1: The phase 3 ENGAGE trialPramod K. Mistry, Michel Peterschmitt, Regina Tayag et al.|American Journal of Hematology|2017Cited by 99