Open-Label Single-Sequence Crossover Study Evaluating Pharmacokinetics, Efficacy, and Safety of Once-Daily Dosing of Nitisinone in Patients with Hereditary Tyrosinemia Type 1-Reference-Cited by-同舟云学术

Open-Label Single-Sequence Crossover Study Evaluating Pharmacokinetics, Efficacy, and Safety of Once-Daily Dosing of Nitisinone in Patients with Hereditary Tyrosinemia Type 1

Published:2017 Issue: Volume: Page:81-88
ISSN:2192-8304
Container-title:JIMD Reports
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Author:

Guffon Nathalie,Bröijersén Anders,Palmgren Ingrid,Rudebeck Mattias,Olsson Birgitta

Publisher

Springer Berlin Heidelberg

Link

http://link.springer.com/content/pdf/10.1007/8904_2017_29

Reference24 articles.

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2. Coleman CI, Limone B, Sobieraj DM et al (2012) Dosing frequency and medication adherence in chronic disease. J Manag Care Pharm 18:527–539

3. De Jesus VR, Adam BW, Mandel D, Cuthbert CD, Matern D (2014) Succinylacetone as primary marker to detect tyrosinemia type I in newborns and its measurement by newborn screening programs. Mol Genet Metab 113:67–75

4. de Laet C, Dionisi-Vici C, Leonard JV et al (2013) Recommendations for the management of tyrosinaemia type 1. Orphanet J Rare Dis 8:8

5. Dhillon KS, Bhandal AS, Aznar CP, Lorey FW, Neogi P (2011) Improved tandem mass spectrometry (MS/MS) derivatized method for the detection of tyrosinemia type I, amino acids and acylcarnitine disorders using a single extraction process. Clin Chim Acta 412:873–879

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1. Clinical utilization of dried blood spot nitisinone (NTBC) and succinylacetone (SA) concentrations in hereditary tyrosinaemia type 1 – A UK centre experience;Annals of Clinical Biochemistry: International Journal of Laboratory Medicine;2020-09-30

2. Inter‐laboratory analytical improvement of succinylacetone and nitisinone quantification from dried blood spot samples;JIMD Reports;2020-04-04

3. Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1;Pediatric Drugs;2019-10-31