Document Detail

New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride.
MedLine Citation:
PMID:  20714359     Owner:  NLM     Status:  PubMed-not-MEDLINE    
Oral administration of sapropterin hydrochloride, recently approved for use by the US Food and Drug Administration and the European Commission, is a novel approach for the treatment of phenylketonuria (PKU), one of the most common inborn errors of metabolism. PKU is caused by an inherited deficiency of the enzyme phenylalanine hydroxylase (PAH), and the pathophysiology of the disorder is related to chronic accumulation of the free amino acid phenylalanine in tissues. Contemporary therapy is based upon restriction of dietary protein intake, which leads to reduction of blood phenylalanine levels. This therapy is difficult to maintain throughout life, and dietary noncompliance is commonplace. Sapropterin dihydrochloride is a synthetic version of tetrahydrobiopterin, the naturally occurring pterin cofactor that is required for PAH-mediated phenylalanine hydroxylation. In a subset of individuals with PAH deficiency, sapropterin administration leads to reduction in blood phenylalanine levels independent of dietary protein. For these individuals, sapropterin is an effective novel therapy for PKU.
Cary O Harding
Related Documents :
6933959 - The occurrence of carcinogenic polycyclic hydrocarbons in foodstuffs in hungary.
24237359 - Impacts of wearing complete dentures on bolus transport during feeding in elderly edent...
12038829 - Occurrence and destruction of pahs, pcbs, ciphs, cibzs, and pcdd/fs in ash from gasific...
18778929 - Size distribution and number concentration of particles at the stack of a municipal was...
16111139 - The effect of diesel fuel sulfur content on particulate matter emissions for a nonroad ...
10085569 - The pm2.5 and pm10 particles in urban areas of taiwan.
15910679 - War and food security in eritrea and ethiopia, 1998-2000.
12616579 - Brain dopamine is associated with eating behaviors in humans.
1431259 - Cholera in piura, peru: a modern urban epidemic.
Publication Detail:
Type:  Journal Article     Date:  2010-08-09
Journal Detail:
Title:  Biologics : targets & therapy     Volume:  4     ISSN:  1177-5491     ISO Abbreviation:  Biologics     Publication Date:  2010  
Date Detail:
Created Date:  2010-08-17     Completed Date:  2011-07-14     Revised Date:  2013-05-29    
Medline Journal Info:
Nlm Unique ID:  101321511     Medline TA:  Biologics     Country:  New Zealand    
Other Details:
Languages:  eng     Pagination:  231-6     Citation Subset:  -    
Departments of Molecular and Medical Genetics and Pediatrics, Oregon Health & Science University, Portland, Oregon, USA.
Export Citation:
APA/MLA Format     Download EndNote     Download BibTex
MeSH Terms

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

Previous Document:  Prevention of rotavirus gastroenteritis in infants and children: rotavirus vaccine safety, efficacy,...
Next Document:  Beals-Hecht syndrome and choroidal neovascularization.