Document Detail


The hyperinsulinism/hyperammonemia syndrome.
MedLine Citation:
PMID:  20936362     Owner:  NLM     Status:  In-Process    
Abstract/OtherAbstract:
The hyperinsulinism/hyperammonemia (HI/HA) syndrome is the second most common form of congenital hyperinsulinism (HI). Children affected by this syndrome have both fasting and protein sensitive hypoglycemia combined with persistently elevated ammonia levels. Gain of function mutations in the mitochondrial enzyme glutamate dehydrogenase (GDH) are responsible for the HI/HA syndrome. GDH is expressed in liver, kidney, brain, and pancreatic beta-cells. Patients with the HI/HA syndrome have an increased frequency of generalized seizures, especially absence-type seizures, in the absence of hypoglycemia. The hypoglycemia of the HI/HA syndrome is well controlled with diazoxide, a KATP channel agonist. GDH has also been implicated in another form of HI, short-chain 3-hydroxyacyl-CoA dehydrogenase (SCHAD) deficiency associated HI. The HI/HA syndrome provides a rare example of an inborn error of intermediary metabolism in which the effect of the mutation on enzyme activity is a gain of function.
Authors:
Andrew A Palladino; Charles A Stanley
Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural    
Journal Detail:
Title:  Reviews in endocrine & metabolic disorders     Volume:  11     ISSN:  1573-2606     ISO Abbreviation:  Rev Endocr Metab Disord     Publication Date:  2010 Sep 
Date Detail:
Created Date:  2010-11-08     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  100940588     Medline TA:  Rev Endocr Metab Disord     Country:  United States    
Other Details:
Languages:  eng     Pagination:  171-8     Citation Subset:  IM    
Affiliation:
The Children's Hospital of Philadelphia, Division of Endocrinology and Diabetes, University of Pennsylvania, School of Medicine, Philadelphia, PA 19104, USA. palladinoa@email.chop.edu
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Descriptor/Qualifier:
Grant Support
ID/Acronym/Agency:
R01 DK 53012/DK/NIDDK NIH HHS; R01 DK 56268/DK/NIDDK NIH HHS; UL1 RR024134/RR/NCRR NIH HHS

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