Literature DB >> 15610810

Prenatal and early postnatal treatment in 3-phosphoglycerate-dehydrogenase deficiency.

T J de Koning1, L W J Klomp, A C C van Oppen, F A Beemer, L Dorland, Iet van den Berg, R Berger.   

Abstract

3-phosphoglycerate-dehydrogenase (3-PGDH) deficiency is an L-serine biosynthesis disorder, characterised by congenital microcephaly, severe psychomotor retardation, and intractable seizures. We report prenatal diagnosis of an affected fetus by DNA mutation analysis. Ultrasound assessment showed a reduction in fetal head circumference from the 75th percentile at 20 weeks' gestation to the 29th percentile at 26 weeks. L-serine was then given to the mother, which resulted in an enlarged fetal head circumference to the 76th percentile at 31 weeks. At birth, the girl's head circumference was normal, and at 48 months' follow-up, her psychomotor development has been unremarkable. 3-PGDH deficiency is an inborn metabolic error that can be successfully treated antenatally.

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Year:  2004        PMID: 15610810     DOI: 10.1016/S0140-6736(04)17596-X

Source DB:  PubMed          Journal:  Lancet        ISSN: 0140-6736            Impact factor:   79.321


  33 in total

Review 1.  Prenatal pharmacotherapy for fetal anomalies: a 2011 update.

Authors:  Lisa Hui; Diana W Bianchi
Journal:  Prenat Diagn       Date:  2011-06-03       Impact factor: 3.050

2.  Neu-Laxova syndrome is a heterogeneous metabolic disorder caused by defects in enzymes of the L-serine biosynthesis pathway.

Authors:  Rocio Acuna-Hidalgo; Denny Schanze; Ariana Kariminejad; Ann Nordgren; Mohamad Hasan Kariminejad; Peter Conner; Giedre Grigelioniene; Daniel Nilsson; Magnus Nordenskjöld; Anna Wedell; Christoph Freyer; Anna Wredenberg; Dagmar Wieczorek; Gabriele Gillessen-Kaesbach; Hülya Kayserili; Nursel Elcioglu; Siavash Ghaderi-Sohi; Payman Goodarzi; Hamidreza Setayesh; Maartje van de Vorst; Marloes Steehouwer; Rolph Pfundt; Birgit Krabichler; Cynthia Curry; Malcolm G MacKenzie; Kym M Boycott; Christian Gilissen; Andreas R Janecke; Alexander Hoischen; Martin Zenker
Journal:  Am J Hum Genet       Date:  2014-08-21       Impact factor: 11.025

3.  L-Serine Deficiency Elicits Intracellular Accumulation of Cytotoxic Deoxysphingolipids and Lipid Body Formation.

Authors:  Kayoko Esaki; Tomoko Sayano; Chiaki Sonoda; Takumi Akagi; Takeshi Suzuki; Takuya Ogawa; Masahiro Okamoto; Takeo Yoshikawa; Yoshio Hirabayashi; Shigeki Furuya
Journal:  J Biol Chem       Date:  2015-04-22       Impact factor: 5.157

4.  A yeast-based complementation assay elucidates the functional impact of 200 missense variants in human PSAT1.

Authors:  Amy Sirr; Russell S Lo; Gareth A Cromie; Adrian C Scott; Julee Ashmead; Mirutse Heyesus; Aimée M Dudley
Journal:  J Inherit Metab Dis       Date:  2020-02-27       Impact factor: 4.982

5.  D-serine influences synaptogenesis in a p19 cell model.

Authors:  Sabine A Fuchs; Martin W Roeleveld; Leo W J Klomp; Ruud Berger; Tom J de Koning
Journal:  JIMD Rep       Date:  2012-02-01

Review 6.  Disorders of phospholipids, sphingolipids and fatty acids biosynthesis: toward a new category of inherited metabolic diseases.

Authors:  F Lamari; F Mochel; F Sedel; J M Saudubray
Journal:  J Inherit Metab Dis       Date:  2012-07-20       Impact factor: 4.982

7.  Neu-Laxova syndrome, an inborn error of serine metabolism, is caused by mutations in PHGDH.

Authors:  Ranad Shaheen; Zuhair Rahbeeni; Amal Alhashem; Eissa Faqeih; Qi Zhao; Yong Xiong; Agaadir Almoisheer; Sarah M Al-Qattan; Halima A Almadani; Noufa Al-Onazi; Badi S Al-Baqawi; Mohammad Ali Saleh; Fowzan S Alkuraya
Journal:  Am J Hum Genet       Date:  2014-05-15       Impact factor: 11.025

Review 8.  L-Serine: a Naturally-Occurring Amino Acid with Therapeutic Potential.

Authors:  J S Metcalf; R A Dunlop; J T Powell; S A Banack; P A Cox
Journal:  Neurotox Res       Date:  2017-09-19       Impact factor: 3.911

Review 9.  New treatment paradigms in neonatal metabolic epilepsies.

Authors:  P L Pearl
Journal:  J Inherit Metab Dis       Date:  2009-02-24       Impact factor: 4.982

Review 10.  Epilepsy and inborn errors of metabolism in children.

Authors:  N I Wolf; A García-Cazorla; G F Hoffmann
Journal:  J Inherit Metab Dis       Date:  2009-07-31       Impact factor: 4.982

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