Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
3
pubmed:dateCreated
1998-12-8
pubmed:abstractText
Enzyme replacement therapy for Gaucher diseases, the most prevalent lysosmal storage disease, was originally approved by the FDA for type I patients and has proven to be both safe and effective in reducing hepatosplenomegaly and improving the hematological parameters. However, the use of enzyme treatment in both neuronopathic forms has heretofore been on an investigational or trial basis, with reports of progression of neurological deterioration even at very high doses. To date, there are no guidelines for clinicians with regard to enzyme replacement therapy in the neuronopathic forms of metabolic diseases. Herein. we discuss strategies derived from the literature vis-à-vis treatment of very premature babies and from the Jewish Halachic point of view. In conclusion. we describe recommendations for the ethical treatment and/or withdrawal of treatment, as well as practical guidelines for dosage regimens, in children with neuronopathic Gaucher disease.
pubmed:keyword
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
E
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Sep
pubmed:issn
0009-9163
pubmed:author
pubmed:issnType
Print
pubmed:volume
54
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
179-84
pubmed:dateRevised
2007-11-15
pubmed:meshHeading
pubmed:year
1998
pubmed:articleTitle
Ethical considerations for enzyme replacement therapy in neuronopathic Gaucher disease.
pubmed:affiliation
Gaucher Clinic, Shaare-Zedek Medical Center, Jerusalem, Israel. zimran@md.huji.ac.il
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't