Literature DB >> 17159114

Dopa-responsive hypersomnia and mixed movement disorder due to sepiapterin reductase deficiency.

Jennifer Friedman1, Keith Hyland, Nenad Blau, Mia MacCollin.   

Abstract

Sepiapterin reductase deficiency (SRD) is a rare, treatable disorder of monoamine metabolism with cognitive delay and l-dopa responsive movement disorder. We describe a patient with SRD and distinctive phenotypic feature of marked hypersomnolence. Our patient showed improvement with therapies directed at both serotonergic and dopaminergic deficiencies. This case illustrates symptoms that characterize the SRD phenotype and demonstrates the importance of systematic treatment trials addressing the various biochemical abnormalities present.

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Year:  2006        PMID: 17159114     DOI: 10.1212/01.wnl.0000247274.21261.b4

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


  18 in total

1.  Sleep and rhythm consequences of a genetically induced loss of serotonin.

Authors:  Smaranda Leu-Semenescu; Isabelle Arnulf; Caroline Decaix; Fathi Moussa; Fabienne Clot; Camille Boniol; Yvan Touitou; Richard Levy; Marie Vidailhet; Emmanuel Roze
Journal:  Sleep       Date:  2010-03       Impact factor: 5.849

2.  Levodopa response reveals sepiapterin reductase deficiency in a female heterozygote with adrenoleukodystrophy.

Authors:  Ronald Thibert; Keith Hyland; Joe Chiles; Steven Steinberg; Florian Eichler
Journal:  JIMD Rep       Date:  2011-09-16

Review 3.  Combining Human and Rodent Genetics to Identify New Analgesics.

Authors:  Alban Latremoliere; Michael Costigan
Journal:  Neurosci Bull       Date:  2017-07-01       Impact factor: 5.203

4.  Whole-genome sequencing for optimized patient management.

Authors:  Matthew N Bainbridge; Wojciech Wiszniewski; David R Murdock; Jennifer Friedman; Claudia Gonzaga-Jauregui; Irene Newsham; Jeffrey G Reid; John K Fink; Margaret B Morgan; Marie-Claude Gingras; Donna M Muzny; Linh D Hoang; Shahed Yousaf; James R Lupski; Richard A Gibbs
Journal:  Sci Transl Med       Date:  2011-06-15       Impact factor: 17.956

Review 5.  Monoamine neurotransmitter disorders--clinical advances and future perspectives.

Authors:  Joanne Ng; Apostolos Papandreou; Simon J Heales; Manju A Kurian
Journal:  Nat Rev Neurol       Date:  2015-09-22       Impact factor: 42.937

Review 6.  Disorders of biopterin metabolism.

Authors:  Nicola Longo
Journal:  J Inherit Metab Dis       Date:  2009-02-09       Impact factor: 4.982

7.  Combined Sepiapterin Reductase and Methylmalonyl-CoA Epimerase Deficiency in a Second Patient: Cerebrospinal Fluid Polyunsaturated Fatty Acid Level and Follow-Up Under L-DOPA, 5-HTP and BH4 Trials.

Authors:  Michel Mazzuca; Marie-Anne Maubert; Léna Damaj; Fabienne Clot; Marylène Cadoudal; Christele Dubourg; Sylvie Odent; Jean François Benoit; Nadia Bahi-Buisson; Laurence Christa; Pascale de Lonlay
Journal:  JIMD Rep       Date:  2015-03-13

Review 8.  Hereditary spastic paraparesis in adults associated with inborn errors of metabolism: a diagnostic approach.

Authors:  F Sedel; B Fontaine; J M Saudubray; O Lyon-Caen
Journal:  J Inherit Metab Dis       Date:  2007-10-22       Impact factor: 4.982

Review 9.  Neuroimaging findings in children with paediatric neurotransmitter diseases.

Authors:  Wang-Tso Lee; Wen-Chin Weng; Shinn-Forng Peng; Kai-Yuan Tzen
Journal:  J Inherit Metab Dis       Date:  2009-05-20       Impact factor: 4.982

10.  Sepiapterin reductase deficiency in a 2-year-old girl with incomplete response to treatment during short-term follow-up.

Authors:  K Kusmierska; E E W Jansen; C Jakobs; K Szymanska; E Malunowicz; D Meilei; B Thony; N Blau; J Tryfon; D Rokicki; E Pronicka; J Sykut-Cegielska
Journal:  J Inherit Metab Dis       Date:  2009-01-07       Impact factor: 4.982

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