| Laronidase treatment of mucopolysaccharidosis I. | |
| | |
MedLine Citation:
|
PMID: 15691212 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
|
The lysosomal storage disorder (LSD) mucopolysaccharidosis type I (MPS I, McKusick 25280, Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome) is caused by a deficiency in the lysosomal enzyme, alpha-L-iduronidase (EC 3.2.1.76). MPS I patients can present within a diverse clinical spectrum, ranging from classical Hurler syndrome to attenuated Scheie syndrome. Laronidase (Aldurazyme) enzyme replacement therapy has been developed as a treatment strategy for MPS I patients and has been approved for clinical practice. Here we review the pre-clinical studies and clinical trials that have been used to demonstrate that intravenous laronidase therapy is well tolerated and effective for treating MPS I patients who do not have neuronal pathology. Current challenges for a viable treatment strategy for all MPS I patients include development of an early screening protocol that identifies patients before the onset of irreversible pathology, methods to predict disease severity, appropriate treatment for neuropathology, and an effective patient monitoring regimen. |
| | |
Authors:
|
Ed J Wraith; John J Hopwood; Maria Fuller; Peter J Meikle; Doug A Brooks |
Publication Detail:
|
Type: Journal Article; Research Support, Non-U.S. Gov't; Review |
Journal Detail:
|
Title: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy Volume: 19 ISSN: 1173-8804 ISO Abbreviation: BioDrugs Publication Date: 2005 |
Date Detail:
|
Created Date: 2005-02-04 Completed Date: 2005-06-14 Revised Date: 2007-11-15 |
Medline Journal Info:
|
Nlm Unique ID: 9705305 Medline TA: BioDrugs Country: New Zealand |
Other Details:
|
Languages: eng Pagination: 1-7 Citation Subset: IM |
Affiliation:
|
Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Manchester, UK. |
Export Citation:
|
APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
|
Adolescent Adult Animals Clinical Trials as Topic Half-Life Humans Iduronidase / adverse effects, pharmacokinetics, therapeutic use* Infusions, Intravenous Mucopolysaccharidosis I / drug therapy*, genetics, metabolism |
| Chemical | |
Reg. No./Substance:
|
EC 3.2.1.76/Iduronidase |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
Previous Document: Look-alike, sound-alike drugs review: include look-alike packaging as an additional safety check.
Next Document: Alemtuzumab in the treatment of chronic lymphocytic leukemia.