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Enzyme replacement therapy for Gaucher disease
Open accessAbstract
Four patients with moderately severe type I Gaucher disease were treated with commercially available mannose terminated glucocerebrosidase (Ceredase; Genzyme, Boston, MA) for up to 13 months. The enzyme was administered at the rate of three to four times weekly at one fourth the total recommended dosage, greatly decreasing the cost. Marked regression of hepatomegaly and improvement in liver function tests, peripheral blood counts, and serum angiotensin- converting enzyme levels were documented. The two patients with pulmonary involvement manifested improvement in pulmonary function tests. Skeletal disease remained unchanged.
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Citations
Cited by
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Protein glycosylation: Structural and functional aspects
LIS, H., & SHARON, N. (1993) · European Journal of Biochemistry · doi:10.1111/j.1432-1033.1993.tb18347.x · Cited by 833
Therapeutic goals in the treatment of Gaucher disease
Pastores, G. M., Weinreb, N. J., Aerts, H., Andria, G., Cox, T. M., Giralt, M., Grabowski, G. A., Mistry, P. K., & Tylki-Szymańska, A. (2004) · Seminars in Hematology · doi:10.1053/j.seminhematol.2004.07.009 · Cited by 422
Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin
Platt, F. M., Neises, G. R., Reinkensmeier, G., Townsend, M. J., Perry, V. H., Proia, R. L., Winchester, B., Dwek, R. A., & Butters, T. D. (1997) · Science · doi:10.1126/science.276.5311.428 · Cited by 340
Gene therapy for lysosomal storage diseases
Sands, M. S., & Davidson, B. L. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.006 · Cited by 285
Metadata
- Authors
- Beutler, E., Kay, A., Saven, A., Garver, P., Thurston, D., Dawson, A., & Rosenbloom, B.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 1991-09-01
- Volume
- 78
- Issue
- 5
- Pages
- 1183-1189
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2256622923
- Cited by
- 169
- References
- 0
- FWCI
- 8.89
- Type
- journal article
- Language
- en
- Open access
- bronze
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- Beutler, E., Kay, A., Saven, A., Garver, P., Thurston, D., Dawson, A., & Rosenbloom, B. (1991). Enzyme replacement therapy for Gaucher disease. Blood. https://doi.org/10.1182/blood.v78.5.1183.1183
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
