Response to Letter to the Editor on “Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options”

Cox TM Drelichman G Cravo R . Eliglustat maintains long-term clinical stability in patient with Gaucher disease type 1 stabilized on enzyme therapy. Blood. 2017;129:2375-2383.

Mistry PK Lukin E Ben Turkia H . Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial. JAMA. 2015;313:695-706.

Puzo J Alfonso P Irun P Gervas J Pocovi M Giraldo P . Changes in the atherogenic profile of patients with type 1 Gaucher disease after miglustat therapy. Atherosclerosis. 2010;209:515-519.

CEREZYME–Imiglucerase Injection, Powder, Lyophilized, for Solution [package insert]. Cambridge, MA: Genzyme Corporation; 2017.

Zimran A Brill-Almon E Chertkoff R . Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011;118(22):5767-5773.

Pastores GM Turkia HB Gonzalez DE . Development of anti-velaglucerase alfa antibodies in clinical trial-treated patients with Gaucher disease. Blood Cells Mol Dis. 2016;59:37-43.

Elstein D Hughes D Goker-Alphan O . Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher disease. J Obstet Gynaecol Res. 2014;40:968-975.

Elstein D Rosenbaum H Chertkoff R Zimran A . Taliglucerase alfa during pregnancy for patients with type 1 Gaucher disease. Mol Genet Metab. 2016;117:S44.

ELELYSO (Taliglucerase Alfa) Injection, Powder, Lyophilized, for Solution. New York, NY: Pfizer Laboratories; 2017.

VPRIV (Velaglucerase Alfa) Injection, Powder, Lyophilized, for Solution. Lexington, MA: Shire US Manufacturing, Inc; 2016.

Food and Drug Administration. Cumulative List of Orphan Drugs Designated and/or Approved. Rockville, MD: Food and Drug Administration; 2017.