@article{hlwoodcock:Schiffmann1997,
	title = {Prospective study of neurological responses to treatment with macrophage-targeted glucocerebrosidase in patients with type 3 Gaucher's disease},
	author = {R. Schiffmann and M. P. Heyes and J. M. Aerts and J. M. Dambrosia and M. C. Patterson and T. Degraba and C. C. Parker and G. C. Zirzow and K. Oliver and G. Tedeschi and R. O. Brady and N. W. Barton and C. Nagineni and C. R. Kaneski and G. J. Murray and J. J. Higgins and A. Tournay and T. K. Banerjee and C. Kreps and L. J. C. Scott and M. A. Mckee and K. Crutchfield and K. Frei},
	journal = {Annal. Neur.},
	month = {OCT},
	pages = {613--621},
	volume = {42},
	year = {1997},
	biburl = {http://www.bibsonomy.org/bibtex/21beca1fa361db94bc4771832b6f4f711/hlwoodcock},
	abstract = {We prospectively evaluated the clinical and biochemical responses to enzyme-replacement therapy (ERT) with macrophage-targeted glucocerebrosidase (Ceredase) infusions in 5 patients (age, 3.5-8.5 years) with type 3 Gaucher's disease. The patients were followed for up to 5 years. Enzyme dosage ranged from 120 to 480 U/kg of body weight/month. Systemic manifestations of the disease regressed in all patients. Neurological deficits remained stable in 3 patients and slightly improved in 1. One patient developed myoclonic encephalopathy. Cognitive deterioration occurred in 1 patient and electroencephalographic deterioration in 2. Sequential cerebrospinal fluid (CSF) samples were obtained during the first 3 years of treatment in 3 patients and were analyzed for biochemical markers of disease burden. Glucocerebroside and psychosine levels were not elevated in these specimens, whereas chitotriosidase and quinolinic acid were elevated in 2 patients. Progressive decrease in the CSF levels of these latter macrophage markers during 3 years of treatment implies a decreased number of Gaucher sells in the cerebral perivascular space. Similar changes were not observed in the patient who had a poor neurological outcome. In conclusion, ERT reverses systemic manifestations of type 3 Gaucher's disease and appears to reduce the burden of Gaucher cells in the brain-CSF compartment in some patients.},
	priority = {2}, citeulike-article-id = {599017},
	keywords = {bibtex-import }
}