Literature DB >> 25343113

Current situation and prospects of newborn screening and treatment for Phenylketonuria in China - compared with the current situation in the United States, UK and Japan.

Lin Mei1, Peipei Song2, Norihiro Kokudo2, Lingzhong Xu3, Wei Tang1.   

Abstract

Phenylketonuria (PKU) is a treat-able and prevent-able inborn error of metabolism which leads to severe mental retardation and neurobehavioral abnormalities. A screening program, especially for early detection, combined with a Phe-restricted therapeutic diet can help to control the process of PKU of most patients. The China government has put more emphasis on newborn screening and treatment against PKU, yet by comparing the situation of newborn screening and treatment against PKU in China and the relatively developed countries - United States, United Kingdom and Japan, the newborn screening and treatment against PKU in China is relatively weak and many deficiencies are found. More studies concerning multi-stage target blood Phe concentration criteria, a policy that requires newborn screening has to be taken, better financial support for newborn screening, publicity for newborn screening, and national guidelines for treatment of PKU may be prospects in China and may provide some support for better development of newborn screening and treatment against PKU in China.

Entities:  

Keywords:  Phenylketonuria (PKU); guidelines; newborn screening; policy; treatment

Year:  2013        PMID: 25343113      PMCID: PMC4204557          DOI: 10.5582/irdr.2013.v2.4.106

Source DB:  PubMed          Journal:  Intractable Rare Dis Res        ISSN: 2186-3644


  52 in total

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Journal:  Lancet       Date:  1953-10-17       Impact factor: 79.321

2.  Intractable and rare diseases research in Asia.

Authors:  Peipei Song; Jianjun Gao; Yoshinori Inagaki; Norihiro Kokudo; Wei Tang
Journal:  Biosci Trends       Date:  2012-04       Impact factor: 2.400

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Authors:  Carmencita D Padilla; Bradford L Therrell
Journal:  J Inherit Metab Dis       Date:  2007-07-23       Impact factor: 4.982

4.  Necessity of cooperation with government on publication of scientific research results for intractable diseases.

Authors:  Yoshinori Inagaki; Peipei Song
Journal:  Intractable Rare Dis Res       Date:  2013-05

5.  Highlights of the new WHO Report on Newborn and Infant Hearing Screening and implications for developing countries.

Authors:  Bolajoko O Olusanya
Journal:  Int J Pediatr Otorhinolaryngol       Date:  2011-06       Impact factor: 1.675

Review 6.  National Institutes of Health Consensus Development Conference Statement: phenylketonuria: screening and management, October 16-18, 2000.

Authors: 
Journal:  Pediatrics       Date:  2001-10       Impact factor: 7.124

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Authors:  P Guldberg; K F Henriksen; I Sipilä; F Güttler; A de la Chapelle
Journal:  J Med Genet       Date:  1995-12       Impact factor: 6.318

8.  Rare disease patients in China anticipate the sunlight of legislation.

Authors:  J J Gao; P P Song; W Tang
Journal:  Drug Discov Ther       Date:  2013-06

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Authors:  F J van Spronsen; K Kiaer Ahring; M Gizewska
Journal:  J Inherit Metab Dis       Date:  2009-01-13       Impact factor: 4.982

10.  Mutations of the phenylalanine hydroxylase gene in patients with phenylketonuria in Shanxi, China.

Authors:  Yong-An Zhou; Yun-Xia Ma; Quan-Bin Zhang; Wei-Hua Gao; Jian-Ping Liu; Jian-Ping Yang; Gai-Xiu Zhang; Xiao-Gang Zhang; Liang Yu
Journal:  Genet Mol Biol       Date:  2012-10-16       Impact factor: 1.771

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  7 in total

1.  Noninvasive prenatal testing in China: Future detection of rare genetic diseases?

Authors:  Lin Mei; Qi Tang; Baiyu Sun; Lingzhong Xu
Journal:  Intractable Rare Dis Res       Date:  2014-08

Review 2.  Newborn screening for Duchenne muscular dystrophy in China: follow-up diagnosis and subsequent treatment.

Authors:  Qing Ke; Zheng-Yan Zhao; Robert Griggs; Veronica Wiley; Anne Connolly; Jennifer Kwon; Ming Qi; Daniel Sheehan; Emma Ciafaloni; R Rodney Howell; Petra Furu; Peter Sazani; Arvind Narayana; Michele Gatheridge
Journal:  World J Pediatr       Date:  2017-05-17       Impact factor: 2.764

3.  Diagnostic and management practices for phenylketonuria in 19 countries of the South and Eastern European Region: survey results.

Authors:  Maria Giżewska; Anita MacDonald; Amaya Bélanger-Quintana; Alberto Burlina; Maureen Cleary; Turgay Coşkun; François Feillet; Ania C Muntau; Friedrich K Trefz; Francjan J van Spronsen; Nenad Blau
Journal:  Eur J Pediatr       Date:  2015-09-08       Impact factor: 3.183

4.  Household financial burden of phenylketonuria and its impact on treatment in China: a cross-sectional study.

Authors:  Lin Wang; Hui Zou; Fang Ye; Kundi Wang; Xiaowen Li; Zhihua Chen; Jie Chen; Bingjuan Han; Weimin Yu; Chun He; Ming Shen
Journal:  J Inherit Metab Dis       Date:  2016-11-10       Impact factor: 4.982

5.  Development of the US English version of the phenylketonuria - quality of life (PKU-QOL) questionnaire.

Authors:  Elaina Jurecki; Amy Cunningham; Vanessa Birardi; Grégory Gagol; Catherine Acquadro
Journal:  Health Qual Life Outcomes       Date:  2017-03-09       Impact factor: 3.186

6.  Benzene Derivatives from Ink Lead to False Positive Results in Neonatal Hyperphenylalaninemia Screening with Ninhydrin Fluorometric Method.

Authors:  Shuren Feng; Joanne Mei; Lu Yang; Ping Luo; Xiaonan Wang; Yuan Wang; Jingyi Yao; Lan Cui; Lei Pan; Zefang Wang; Li Xin
Journal:  Int J Neonatal Screen       Date:  2020-02-29

7.  CRISPR/Cas9 generated knockout mice lacking phenylalanine hydroxylase protein as a novel preclinical model for human phenylketonuria.

Authors:  Kuldeep Singh; Cathleen S Cornell; Robert Jackson; Mostafa Kabiri; Michael Phipps; Mitul Desai; Robert Fogle; Xiaoyou Ying; Gulbenk Anarat-Cappillino; Sarah Geller; Jennifer Johnson; Errin Roberts; Katie Malley; Tim Devlin; Matthew DeRiso; Patricia Berthelette; Yao V Zhang; Susan Ryan; Srinivas Rao; Beth L Thurberg; Dinesh S Bangari; Sirkka Kyostio-Moore
Journal:  Sci Rep       Date:  2021-03-31       Impact factor: 4.379

  7 in total

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