Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications

Author:

van Dussen Laura,Biegstraaten Marieke,Dijkgraaf Marcel GW,Hollak Carla EM

Publisher

Springer Science and Business Media LLC

Subject

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

Reference61 articles.

1. Grabowski GA, Petsko GA, Kolodny EH: Gaucher Disease. The Online Metabolic & Molecular Bases of Inherited Disease. Edited by: Valle D, Beaudet AL, Vogelstein B, Kinzler K, Antonarakis SE, Ballabio A. New York: McGraw-Hill, 2010.

2. Kolodny EH, Ullman MD, Mankin HJ, Raghavan SS, Topol J, Sullivan JL: Phenotypic manifestations of Gaucher disease: clinical features in 48 biochemically verified type 1 patients and comment on type 2 patients. ProgClinBiolRes. 1982, 95: 33-65.

3. de Fost M, vom Dahl S, Weverling GJ, Brill N, Brett S, Haussinger D, Hollak CE: Increased incidence of cancer in adult Gaucher disease in Western Europe. Blood Cells Mol Dis. 2006, 36: 53-58. 10.1016/j.bcmd.2005.08.004.

4. Shiran A, Brenner B, Laor A, Tatarsky I: Increased risk of cancer in patients with Gaucher disease. Cancer. 1993, 72: 219-224. 10.1002/1097-0142(19930701)72:1<219::AID-CNCR2820720139>3.0.CO;2-Y.

5. Neudorfer O, Giladi N, Elstein D, Abrahamov A, Turezkite T, Aghai E, Reches A, Bembi B, Zimran A: Occurrence of Parkinson’s syndrome in type I Gaucher disease. QJM. 1996, 89: 691-694. 10.1093/qjmed/89.9.691.

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