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

[1] Stirnemann J(2011)Comite d'evaluation du traitement de la maladie de Gaucher. [Gaucher disease] Rev Prat 61 165-8

[2] de Villemeur TB(2000)The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease Arch Intern Med 160 2835-43

[3] Heraoui D(1993)Gaucher disease. Enzymology, genetics, and treatment Adv Hum Genet 21 377-441

[4] Belmatoug N(2006)The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis Arch Pediatr Adolesc Med 160 603-8

[5] Charrow J(2009)[Management of Gaucher disease in France] Presse Med 38 2S1-5

[6] Andersson HC(2013)Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment J Inherit Metab Dis 36 543-53

[7] Kaplan P(2001)Clinically relevant therapeutic endpoints in type I Gaucher disease J Inherit Metab Dis 24 97-105

[8] Kolodny EH(2008)A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase Am J Hematol 83 890-5

[9] Mistry P(2009)Dose–response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1 Genet Med 11 92-100

[10] Pastores G(2004)Therapeutic goals in the treatment of Gaucher disease Semin Hematol 41 4-14

[1] Stirnemann J(2011)Comite d'evaluation du traitement de la maladie de Gaucher. [Gaucher disease] Rev Prat 61 165-8

[2] de Villemeur TB(2000)The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease Arch Intern Med 160 2835-43

[3] Heraoui D(1993)Gaucher disease. Enzymology, genetics, and treatment Adv Hum Genet 21 377-441

[4] Belmatoug N(2006)The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis Arch Pediatr Adolesc Med 160 603-8

[5] Charrow J(2009)[Management of Gaucher disease in France] Presse Med 38 2S1-5

[6] Andersson HC(2013)Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment J Inherit Metab Dis 36 543-53

[7] Kaplan P(2001)Clinically relevant therapeutic endpoints in type I Gaucher disease J Inherit Metab Dis 24 97-105

[8] Kolodny EH(2008)A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase Am J Hematol 83 890-5

[9] Mistry P(2009)Dose–response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1 Genet Med 11 92-100

[10] Pastores G(2004)Therapeutic goals in the treatment of Gaucher disease Semin Hematol 41 4-14