2022
Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trials
Cassiman D, Mistry P, Jones S, Lachmann R, Lukina E, Prada C, Thurberg B, Wasserstein M, Foster M, Patel R, Ribes M, Underhill L, Peterschmitt M. Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trials. Molecular Genetics And Metabolism 2022, 135: s29. DOI: 10.1016/j.ymgme.2021.11.058.Peer-Reviewed Original Research
2020
Individual patient responses to eliglustat in treatment-naïve adults with Gaucher disease type 1: Final data from the phase 3 ENGAGE trial
Mistry P, Lukina E, Turkia H, Shankar S, Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster M, Gaemers S, Peterschmitt M. Individual patient responses to eliglustat in treatment-naïve adults with Gaucher disease type 1: Final data from the phase 3 ENGAGE trial. Molecular Genetics And Metabolism 2020, 129: s110-s111. DOI: 10.1016/j.ymgme.2019.11.284.Peer-Reviewed Original Research
2019
Effects of oral eliglustat on skeletal manifestations in patients with type 1 Gaucher disease: Results from four completed clinical trials after long-term treatment
Cox T, Charrow J, Lukina E, Mistry P, Marinakis T, Foster M, Gaemers S, Peterschmitt M. Effects of oral eliglustat on skeletal manifestations in patients with type 1 Gaucher disease: Results from four completed clinical trials after long-term treatment. Molecular Genetics And Metabolism 2019, 126: s43. DOI: 10.1016/j.ymgme.2018.12.091.Peer-Reviewed Original Research
2013
Rare disease clinical research network's urea cycle consortium delivers a successful clinical trial to improve alternate pathway therapy
Mistry PK. Rare disease clinical research network's urea cycle consortium delivers a successful clinical trial to improve alternate pathway therapy. Hepatology 2013, 57: 2100-2102. PMID: 23080135, DOI: 10.1002/hep.26106.Peer-Reviewed Original Research