Morbus Gaucher
Author:
Publisher
Elsevier
Reference22 articles.
1. Belmatoug N, Di Rocco M, Fraga C et al.: Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe. Eur J Intern Med 37 (2017) 25–32.
2. Brand M, Muller A, Alsop J et al.: Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) in miglustat (Zavesca®)-treated patients. Pharmacoepidemiol Drug Saf 24(3) (2015) 329–333.
3. Burton BK, Ellis AG, Orr B et al.: Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab 134(1–2) (2021) 182–187.
4. Grabowski GA, Kacena K, Cole JA et al.: Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. Genet Med 11(2) (2009) 92–100.
5. Hollak CE, Belmatoug N, Cole JA et al.: Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4–5 years. Br J Haematol 158(4) (2012) 528–538.
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