About: Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria

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About: Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria Goto Sponge NotDistinct Permalink

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scientific article published on 01 November 2008

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rdf:type	Item
description	wetenschappelijk artikel (nl) article scientifique publié en 2008 (fr) наукова стаття, опублікована в листопаді 2008 (uk) im November 2008 veröffentlichter wissenschaftlicher Artikel (de) artículu científicu espublizáu en payares de 2008 (ast) scientific article published on 01 November 2008 (en) գիտական հոդված հրատարակված 2008 թվականի նոյեմբերի 1-ին (hy)
publication date	wds:Q79644250-F49CF684-A716-450C-B543-F7DB249D9A48
publication date	2008-11-01 00:00:00Z (xsd:dateTime)
author name string	wds:Q79644250-0BD9D143-98C3-4802-BB6D-6F507CC6C47E wds:Q79644250-1B6B5C97-DD32-42EB-8263-18E44FE134D8 wds:Q79644250-438CA4CD-3255-456B-BB7D-CF04920CD62F wds:Q79644250-4F5E9F6C-F945-445D-ACBA-2B15375EB2CB wds:Q79644250-6BE7F900-6751-477B-B23F-42A84F00CEB1 wds:Q79644250-7B5E0E6E-BD91-4518-A1D3-E0A2BF6DB5AA wds:Q79644250-9FFDDFF2-2D69-48A8-B85B-76E0A5EB4E80 wds:Q79644250-A26550F2-F141-4D72-8DAE-465B745C992D wds:Q79644250-A403A016-BDD6-43CF-A116-E74A9A2E7206 wds:Q79644250-A6C2479B-FB90-4982-8573-CADED9A6E549 wds:Q79644250-BBE7216A-D9DA-43F7-B538-4BA488885FC6 wds:Q79644250-BDA2003D-BE86-4F0F-BA9A-A84474A9553A wds:Q79644250-C573A816-8663-4494-ADA5-9A7013AECED5 wds:Q79644250-CF9CA86E-0389-4243-B6B7-0826F37AE9C8 wds:Q79644250-D57FB84E-59A4-4C37-9204-FFA7395C4414
author name string	Marcello Giovannini Udo Wendel Jon Wolff Phillip Lee Melissa Wasserstein Alex Dorenbaum Barbara K Burton Heidi Christ-Schmidt Judith Bebchuk Andrew A Morris Eileen P Treacy Eric Crombez Lewis Waber Margretta Seashore Sapropterin Research Group
rdfs:label	Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (en) Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (nl)
skos:prefLabel	Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (en) Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (nl)
name	Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (en) Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (nl)
title	wds:Q79644250-17C1F928-7A86-4D26-9508-D02DE7B39709
title	Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria (en)
page(s)	wds:Q79644250-E87C4FEA-EB4B-42A1-AFAE-B38F1F46B64E
page(s)	2851-2859
instance of	wds:Q79644250-A1A01B9A-E3E7-4AF4-A9EB-DE3E4C92933C
instance of	scholarly article
main subject	wds:Q79644250-67F4BA90-8AB9-465A-9AFE-311E22321CE7
main subject	sapropterin
PubMed ID	wds:Q79644250-7BE56A0A-E437-4E94-915D-17283DDFBB3F
PubMed ID	http://rdf.ncbi.nlm.nih.gov/pubchem/reference/18932221
PubMed ID	18932221
published in	wds:Q79644250-AF4F4C72-6487-4D98-B030-1A0C1FB683B9
published in	American Journal of Medical Genetics
issue	wds:Q79644250-E607C65A-305B-4D94-AA6E-19E1E4DFFA2E
volume	wds:Q79644250-76881E5B-D79C-4656-8F62-C551EC8B2B31
issue	22
volume	146A
DOI	wds:Q79644250-58B93D97-8566-4FF6-8126-BA11B14E9811
DOI	http://dx.doi.org/10.1002/AJMG.A.32562
DOI	10.1002/AJMG.A.32562
is about of	https://www.wikidata.org/wiki/Special:EntityData/Q79644250
is cites work of	Achieving the "triple aim" for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence framework New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride 5-Hydroxytryptophan during critical postnatal period improves cognitive performances and promotes dendritic spine maturation in genetic mouse model of phenylketonuria. Innovative strategies to treat protein misfolding in inborn errors of metabolism: pharmacological chaperones and proteostasis regulators. Phenylketonuria Suboptimal outcomes in patients with PKU treated early with diet alone: revisiting the evidence Optimizing the use of sapropterin (BH(4)) in the management of phenylketonuria. Large neutral amino acids supplementation in phenylketonuric patients. BH(4) therapy impacts the nutrition status and intake in children with phenylketonuria: 2-year follow-up Sapropterin therapy increases stability of blood phenylalanine levels in patients with BH4-responsive phenylketonuria (PKU). Sapropterin dihydrochloride for phenylketonuria Up to date knowledge on different treatment strategies for phenylketonuria Adjusting diet with sapropterin in phenylketonuria: what factors should be considered? Pharmacokinetics of sapropterin in patients with phenylketonuria Long-term follow-up of patients with phenylketonuria receiving tetrahydrobiopterin treatment Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: results of a phase 3b study Phenylketonuria Scientific Review Conference: state of the science and future research needs Influence of PAH Genotype on Sapropterin Response in PKU: Results of a Single-Center Cohort Study Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry Tetrahydrobiopterin (BH4) in PKU: effect on dietary treatment, metabolic control, and quality of life Tetrahydrobiopterin responsiveness after extended loading test of 12 Danish PKU patients with the Y414C mutation Long-term treatment with tetrahydrobiopterin in phenylketonuria: treatment strategies and prediction of long-term responders. Individualized long-term outcomes in blood phenylalanine concentrations and dietary phenylalanine tolerance in 11 patients with primary phenylalanine hydroxylase (PAH) deficiency treated with Sapropterin-dihydrochloride. Sapropterin dihydrochloride for phenylketonuria Fluctuations in phenylalanine concentrations in phenylketonuria: a review of possible relationships with outcomes. Sapropterin: a review of its use in the treatment of primary hyperphenylalaninaemia Long-term safety and efficacy of sapropterin: the PKUDOS registry experience Long-term follow-up of patients with phenylketonuria treated with tetrahydrobiopterin: a seven years experience A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria A Systematic Review of BH4 (Sapropterin) for the Adjuvant Treatment of Phenylketonuria Inherited Metabolic Disorders: Aspects of Chronic Nutrition Management Phenylketonuria as a model for protein misfolding diseases and for the development of next generation orphan drugs for patients with inborn errors of metabolism Use of Gastrostomy Tube to Prevent Maternal PKU Syndrome

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