Document Detail


Long-term follow-up of well-treated nephropathic cystinosis patients.
MedLine Citation:
PMID:  15480385     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
We report the excellent clinical outcomes of siblings with nephropathic cystinosis treated diligently with cysteamine starting at 20 months and 2 months of age. Now 15 and 8 years old, they have glomerular filtration rates of 78 and 105 mL/min/1.73m 2 , respectively. These cases illustrate the critical importance of early diagnosis and treatment.
Authors:
Robert Kleta; Isa Bernardini; Masako Ueda; William S Varade; Chanika Phornphutkul; Donna Krasnewich; William A Gahl
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Publication Detail:
Type:  Case Reports; Journal Article    
Journal Detail:
Title:  The Journal of pediatrics     Volume:  145     ISSN:  0022-3476     ISO Abbreviation:  J. Pediatr.     Publication Date:  2004 Oct 
Date Detail:
Created Date:  2004-10-13     Completed Date:  2004-12-07     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  0375410     Medline TA:  J Pediatr     Country:  United States    
Other Details:
Languages:  eng     Pagination:  555-60     Citation Subset:  AIM; IM    
Affiliation:
Section on Human Biochemical Genetics, Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20892-1851, USA. kletar@mail.nih.gov <kletar@mail.nih.gov>
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MeSH Terms
Descriptor/Qualifier:
Cysteamine / administration & dosage*
Cystine / metabolism
Fanconi Syndrome / drug therapy*
Female
Follow-Up Studies
Glomerular Filtration Rate
Growth Disorders / prevention & control
Humans
Infant
Infant, Newborn
Male
Time Factors
Treatment Outcome
Chemical
Reg. No./Substance:
56-89-3/Cystine; 60-23-1/Cysteamine
Comments/Corrections
Comment In:
J Pediatr. 2004 Oct;145(4):436-8   [PMID:  15480361 ]

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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