Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1

[11] Grabowski GA(2002)Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry Am J Med 113 112-9

[12] Kaplan P(2005)International Collaborative Gaucher Group USRC: Individualization of long-term enzyme replacement therapy for Gaucher disease Genet Med 7 105-10

[13] Andersson HC(2006)Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis Blood 108 830-5

[14] Kacena KA(2007)Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease J Bone Miner Res 22 119-26

[15] Yee JD(2008)Imiglucerase and its use for the treatment of Gaucher's disease Expert Opin Pharmacother 9 1987-2000

[16] Stirnemann J(2010)Force Majeure: Therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease Blood Cells Mol Dis 44 41-7

[17] Belmatoug N(2012)The French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients Orphanet J Rare Dis 7 77-4

[18] Weinreb NJ(2011)Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply Blood Cells Mol Dis 46 111-22

[19] Goldblatt J(2013)Did the temporary shortage in supply of imiglucerase have clinical consequences? Retrospective observational study on 34 italian Gaucher type I patients JIMD Rep 7 117-4

[20] Villalobos J(2012)Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes Haematologica 97 1850-8

[11] Grabowski GA(2002)Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry Am J Med 113 112-9

[12] Kaplan P(2005)International Collaborative Gaucher Group USRC: Individualization of long-term enzyme replacement therapy for Gaucher disease Genet Med 7 105-10

[13] Andersson HC(2006)Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis Blood 108 830-5

[14] Kacena KA(2007)Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease J Bone Miner Res 22 119-26

[15] Yee JD(2008)Imiglucerase and its use for the treatment of Gaucher's disease Expert Opin Pharmacother 9 1987-2000

[16] Stirnemann J(2010)Force Majeure: Therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease Blood Cells Mol Dis 44 41-7

[17] Belmatoug N(2012)The French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients Orphanet J Rare Dis 7 77-4

[18] Weinreb NJ(2011)Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply Blood Cells Mol Dis 46 111-22

[19] Goldblatt J(2013)Did the temporary shortage in supply of imiglucerase have clinical consequences? Retrospective observational study on 34 italian Gaucher type I patients JIMD Rep 7 117-4

[20] Villalobos J(2012)Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes Haematologica 97 1850-8