Long-term safety and efficacy of sapropterin: The PKUDOS registry experience-Reference-Cited by-同舟云学术

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Long-term safety and efficacy of sapropterin: The PKUDOS registry experience

Published:2015-04 Issue:4 Volume:114 Page:557-563
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Longo Nicola,Arnold Georgianne L.,Pridjian Gabriella,Enns Gregory M.,Ficicioglu Can,Parker Susan,Cohen-Pfeffer Jessica L.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference32 articles.

1. National Institutes of Health Consensus Development Conference Statement: phenylketonuria: screening and management, October 16–18, 2000;National Institutes of Health Consensus Development Panel;Pediatrics,2001

2. PAHdb: a locus-specific knowledgebase;Scriver;Hum. Mutat.,2000

3. For the American College of Medical Genetics and Genomics Therapeutic Committee, phenylalanine hydroxylase deficiency diagnosis and management guideline;Vockley;Genet. Med.,2014

4. Recommendations for the nutrition management of phenylalanine hydroxylase deficiency;Singh;Genet. Med.,2014

5. Phenylalanine blood levels and clinical outcomes in phenylketonuria: a systematic literature review and meta-analysis;Waisbren;Mol. Genet. Metab.,2007

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1. Long‐term safety of sapropterin in paediatric and adult individuals with phenylalanine hydroxylase deficiency: Final results of the Kuvan® Adult Maternal Paediatric European Registry multinational observational study;Journal of Inherited Metabolic Disease;2024-09-05

2. Longitudinal Dietary Intake Data in Patients with Phenylketonuria from Europe: The Impact of Age and Phenylketonuria Severity;Nutrients;2024-08-31

3. Blood Phenylalanine Levels in Patients with Phenylketonuria from Europe between 2012 and 2018: Is It a Changing Landscape?;Nutrients;2024-06-28

4. Long-term comparative effectiveness of pegvaliase versus medical nutrition therapy with and without sapropterin in adults with phenylketonuria;Molecular Genetics and Metabolism;2024-01

5. The treatment of biochemical genetic diseases: From substrate reduction to nucleic acid therapies;Molecular Genetics and Metabolism;2023-11

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