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

Author:

Stirnemann Jérôme,Rose Christian,Serratrice Christine,Dalbies Florence,Lidove Olivier,Masseau Agathe,Pers Yves-Marie,Baron Camille,Belmatoug Nadia

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Reference34 articles.

1. Beutler E, Grabowski G. Glucosylceramide lipidosis–Gaucher disease. In: Scriver C, Beaudet A, Sly W, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York: McGraw-Hill Publishers; 2001. p. 3635–68.

2. Stirnemann J, de Villemeur TB, Heraoui D, Belmatoug N. Comite d'evaluation du traitement de la maladie de Gaucher. [Gaucher disease]. Rev Prat. 2011;61:165–8.

3. Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, et al. The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease. Arch Intern Med. 2000;160:2835–43.

4. Grabowski GA. Gaucher disease. Enzymology, genetics, and treatment. Adv Hum Genet. 1993;21:377–441.

5. Kaplan P, Andersson HC, Kacena KA, Yee JD. The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis. Arch Pediatr Adolesc Med. 2006;160:603–8.

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