Skip to main content
SpringerLink
Log in

Alglucerase for Gaucher’s Disease

Dose, Costs and Benefits

  • Guest Editorial
  • Published:
PharmacoEconomics Aims and scope Submit manuscript

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

References

  • Achord DT, Brot FE, Bell CE, Sly WS. Human beta-glucuronidase: in vivo c1earance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cells. Cell 15: 269–278, 1978

    Article  PubMed  CAS  Google Scholar 

  • Barton NW, Brady R, Dambrosia JM. Treatment of Gaucher’s disease. New England Journal of Medicine 328: 1564–1565, 1993

    Article  PubMed  CAS  Google Scholar 

  • Barton NW, Brady RO, Dambrosia JM, et al. Replacement therapy for inherited enzyme deficiency - macrophage-targeted glucocerebrosidase for Gaucher’s di sease. New England Journal of Medicine 324: 1464–1410, 1991a

    Article  PubMed  CAS  Google Scholar 

  • Barton NW, Brady RO, Murray GJ, et al. Enzyme-replacement therapy for Gaucher’s disease: reply. New England Journal of Medicine 325: 1811, 1991b

    Article  Google Scholar 

  • Barton NW, Furbish FS, Murray GJ, Garfield M, Brady RO. Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proceedings of the National Academy of Sciences of the United States of America 87: 1913–1916, 1990

    Article  PubMed  CAS  Google Scholar 

  • Beutler E. Gaucher disease: new molecular approaches to diagnosis and treatment. Science 256: 794–199, 1992

    Article  PubMed  CAS  Google Scholar 

  • Beutler E, Dale GL, Guinto E, Kuhl W. Enzyme replacement therapy in Gaucher’s disease: preliminary clinical lrial of a new enzyme preparation. Proceedings of the National Academy of Sciences of the United States of America 74: 4620–4623, 1977

    Article  PubMed  CAS  Google Scholar 

  • Beutler E, Figueroa M, Koziol J. Treatment of Gaucher’s disease. New England Journal of Medicine 328: 1567, 1993a

    Google Scholar 

  • Beutler E, Gelbart T. Gaucher disease mutations in non-Jewish patients. British Journal of Haematology, in press, 1993

    Google Scholar 

  • Beutler E, Nguyen NJ, Henneberger MW, et al. Gaucher disease: gene frequencies in the Ashkenazi Jewish population. American Journal of Human Genetics 52: 85–88, 1993b

    PubMed  CAS  Google Scholar 

  • Beutler E, Kay A, Saven A. et al. Enzyme replacement therapy for Gaucher disease. Blood 78: 1183–1189, 1991

    PubMed  CAS  Google Scholar 

  • Brady RO, Pentchev PG, Gal AE, Hibbert SR, Dekaban AS. Replacement therapy for inherited enzyme deficiency. Use of purified glucocerebrosidase in Gaucher’s disease. New England Journal of Medicine 291: 989–993, 1974

    CAS  Google Scholar 

  • Culbertson VL, Rhodes RS, Hill EP, Rhodes PJ. Impact of home infusion therapy on the Colorado Medicaid program budget. American Journal of Hospital Pharmacy 45: 1346–1349, 1988

    PubMed  CAS  Google Scholar 

  • De Duve C. From cytases to Iysosomes. Federal Proceedings 23: 1045–1049, 1964

    Google Scholar 

  • Doebber TW, Wu MS, Bugianesi RL, ct al. Enhanced macrophage uptake of synthetieally glycosylated human placental beta-glucocerebrosidase. Journal of Biological Chemistry 257: 2193–2199, 1982

    PubMed  CAS  Google Scholar 

  • Eto Y, Kawame H, Hasegawa Y, Ohashi T, Ida H, et al. Molecular characteristics in Japanese patients with lipidosis: novel mutations in metachromatic leukodystrophy and Gaucher disease. Molecular and Cellular Biochemistry 119: 179–184, 1993

    Article  PubMed  CAS  Google Scholar 

  • Fallet S, Sibille A, Mendelson R, Shapiro D, Hermann G, et al. Enzyme augmentation in moderate to life-threatening Gaucher disease. Pediatric Research 31: 496–502, 1992

    PubMed  CAS  Google Scholar 

  • Figueroa ML, Rosenbloom BE, Kay AC, et al. A less costly regimen of alglucerase to treat Gaucher’s disease. New England Journal of Medicine 327: 1632–1636, 1992

    Article  PubMed  CAS  Google Scholar 

  • Forster J, Chambers JP, Peters SP, Lee RE, Glew RH. Acute neuropathic Gaucher disease in a black infant. Journal of Pediatrics 93: 823–824, 1978

    Article  PubMed  CAS  Google Scholar 

  • Furbish FS, Oliver KL, Zirzow GC, Brady RO, Barranger JA. Interaction of human placental glucocerebrosidase with hepatic lectins. In Barranger and Brady (Eds). Molecular basis of lysosomal storage disorders, pp.219-232, Academic Press Inc., New York, 1984

    Google Scholar 

  • Garber AM, Clarke AE, Goldman DP, Gluck ME. Federal and private roles in the development and provision of alglucerase therapy for Gaucher disease. US Congress, Office of Technology Assessment, US Government Printing Office, Washington DC, 1992

    Google Scholar 

  • Hollak CEM, Aerts JMFG, van Oers MHJ. Treatment of Gaucher’s disease. New England Journal of Medicine 328: 1565–1566, 1993

    PubMed  CAS  Google Scholar 

  • Kay AC, Saven A, Garver P, Thurston DW, Rosenbloom BF, et al. Enzyme replacement therapy in type I Gaucher disease. Transactions of the Association of American Physicians 104: 258–264, 1991

    PubMed  CAS  Google Scholar 

  • Muguti GI, Nijland A, Gwavava NJT, Nkanza NK. Gaucher’s disease in Zimbabwe. Central African Journal of Medicine 33: 267–269, 1987

    PubMed  CAS  Google Scholar 

  • Murray GJ, Doebber TW, Shen TY, et al. Targeting of synthetically glycOsylated human placental glucocerebrosidase. Biochemical Medicine 34: 241–246, 1985

    Article  PubMed  CAS  Google Scholar 

  • Pastores GM, Sibille AR, Grabowski GA. Enzyme therapy in Gaucher disease type 1: dosage efficacy and adverse effects in thirty three patients treated for 6 to 24 months. Blood 82: 408–416, 1993

    PubMed  CAS  Google Scholar 

  • Sato Y, Beutler E, Binding, internalization, and degradation of mannose-terminated glucocerebrosidase by macrophages. Journal of Clinical Investigation 91: 1909–1917, 1993

    Article  PubMed  CAS  Google Scholar 

  • Williams DN, Bosch D, Boots J, Scheider J. Safety, efficacy, and cost savings in an outpatient intravenous anlibiOlic program. Clinical Therapeutics 15: 169–179, 1993

    PubMed  CAS  Google Scholar 

  • Zimran A, Hollak CEM, Abrahamov A, van Oers MHJ, Kelly M, et al. Home treatment wilh intravenous enzyme replacement therapy for Gaucher disease: an international collaborative stUdy of 33 patients. Blood 82: 1107–1109, 1993

    PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Department of Molecular and Experimental Medicine, The Scripps Research Institute, 10666 North Torrey Pines Road, La Jolla, California, 92037, USA

    Ernest Beutler

  2. Palo Alto Department of Veterans Affairs Medical Center and Division of General Internal Medicine, Stanford University School of Medicine, Stanford, California, USA

    Alan M. Garber

Authors
  1. Ernest Beutler
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Alan M. Garber
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Beutler, E., Garber, A.M. Alglucerase for Gaucher’s Disease. Pharmacoeconomics 5, 453–459 (1994). https://doi.org/10.2165/00019053-199405060-00001

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.2165/00019053-199405060-00001

Search

Navigation