Treatment options for patients with gaucher disease
Joint Authors
Shawqi, Rabah M.
al-Sayyid, Sulaf M.
Source
The Egyptian Journal of Medical Human Genetics
Issue
Vol. 17, Issue 3 (31 Jul. 2016), pp.281-285, 5 p.
Publisher
Egyptian Society of Human Genetics
Publication Date
2016-07-31
Country of Publication
Egypt
No. of Pages
5
Main Subjects
Pharmacy, Health & Medical Sciences
Abstract EN
Gaucher disease is the most common lysosomal storage disorder due to deficiency of ß-glucocerebrosidase.
Since the introduction of Ceredase in 1991, enzyme replacement therapy has been the mainstay of treatment with its major disadvantage of long life dependency on biweekly IV therapy.
It was more than a decade later when the substrate reduction therapy – an oral treatment – was approved for Gaucher disease.
Future therapeutic modalities will include pharmacological chaperon and possibly gene therapy.
The aim of this review is to high light the current and future treatment options for patients with Gaucher disease and to compare their effects and side effects
American Psychological Association (APA)
Shawqi, Rabah M.& al-Sayyid, Sulaf M.. 2016. Treatment options for patients with gaucher disease. The Egyptian Journal of Medical Human Genetics،Vol. 17, no. 3, pp.281-285.
https://search.emarefa.net/detail/BIM-734015
Modern Language Association (MLA)
Shawqi, Rabah M.& al-Sayyid, Sulaf M.. Treatment options for patients with gaucher disease. The Egyptian Journal of Medical Human Genetics Vol. 17, no. 3 (Jul. 2016), pp.281-285.
https://search.emarefa.net/detail/BIM-734015
American Medical Association (AMA)
Shawqi, Rabah M.& al-Sayyid, Sulaf M.. Treatment options for patients with gaucher disease. The Egyptian Journal of Medical Human Genetics. 2016. Vol. 17, no. 3, pp.281-285.
https://search.emarefa.net/detail/BIM-734015
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references : p. 284-285
Record ID
BIM-734015