Treatments fashioned specifically for rare diseases are necessarily very expensive. A fully satisfactory solution to this problem has not yet been found.
This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Registries for orphan drugs: generating evidence or marketing tools?
Orphanet Journal of Rare Diseases Open Access 03 September 2020
-
Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
Orphanet Journal of Rare Diseases Open Access 16 April 2011
Access options
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Gaucher disease: Current issues in diagnosis and treatment. Natl. Inst. Health Technol. Assess. Statement. February 27–March 1, 1–27 (1995).
Robinson, M.L. & Craig, C. Company retires $100M debt. Genzyme's Ceredase hits financial constraints. BioWorld Today 7, 1 (1996).
Zimran, A. et al. Low-dose enzyme replacement therapy for Gaucher's disease: Effects of age, sex, genotype, and clinical features on response to treatment. Am. J. Med. 97, 3–13 (1994).
Balicki, D. & Beutler, E. Gaucher disease. Medicine (Baltimore) 74, 305–323 (1995).
Beutler, E. Treatment regimens in Gaucher's disease. Lancet 346, 581–582 (1995).
Beutler, E. et al. The clinical course of treated and untreated Gaucher disease. A study of 45 patients. Blood Cells Mol. Dis. 21, 86–108 (1995).
Hollak, C.E.M. et al. Individualised low-dose alglucerase therapy for type 1 Gaucher's disease. Lancet 345, 1474–1478 (1995).
Cohen, Y., Elstein, D., Abrahamov, A., Hirsch, H. & Zimran, A. HCG contamination of alglucerase: Clinical implications in low-dose regimen. Am. J. Hematol. 47, 235–236 (1994).
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Beutler, E. The cost of treating Gaucher disease. Nat Med 2, 523–524 (1996). https://doi.org/10.1038/nm0596-523
Issue Date:
DOI: https://doi.org/10.1038/nm0596-523
This article is cited by
-
Registries for orphan drugs: generating evidence or marketing tools?
Orphanet Journal of Rare Diseases (2020)
-
Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
Orphanet Journal of Rare Diseases (2011)
-
Gaucher's treatment: First things first
Nature Medicine (1996)