Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:dateCreated
2003-3-3
pubmed:abstractText
Clinical experience for more than two decades has shown that allogeneic HCT may benefit some but not all patients with inherited metabolic diseases. The HCT procedure is most effective in presymptomatic patients and those with indolent forms of storage diseases but is ineffective in those with overt neurological symptoms or aggressive neonatal or infantile forms. HCT alone does not correct skeletal dysplasia in MPSs and may not prevent development or progression of the peripheral neuropathy in sphingolipidoses and ALD. Decisions regarding HCT in patients with storage diseases should be made by investigators knowledgeable about these diseases, with judicious use of laboratory and clinical resources necessary to reach the best therapeutic decision for the individual patient.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:status
MEDLINE
pubmed:issn
0939-5555
pubmed:author
pubmed:issnType
Print
pubmed:volume
81 Suppl 2
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
S16-9
pubmed:dateRevised
2005-11-16
pubmed:meshHeading
pubmed:year
2002
pubmed:articleTitle
Allogeneic hematopoietic cell transplantation for inborn metabolic diseases.
pubmed:affiliation
Departments of Medicine and Pediatrics, University of Pittsburgh Cancer Institute, University of Pittsburgh, Pittsburgh, Pennsylvania, USA.
pubmed:publicationType
Journal Article, Review