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Journal of Inherited Metabolic Disease

Ausgabe 5/2016

Inhalt (20 Artikel)

Highlights

News and views

Sander M. Houten

Highlights

News and views

Peter Witters

SSIEM 2015

Antenatal manifestations of inborn errors of metabolism: autopsy findings suggestive of a metabolic disorder

Sophie Collardeau-Frachon, Marie-Pierre Cordier, Massimiliano Rossi, Laurent Guibaud, Christine Vianey-Saban

SSIEM 2015

Antenatal manifestations of inborn errors of metabolism: biological diagnosis

Christine Vianey-Saban, Cécile Acquaviva, David Cheillan, Sophie Collardeau-Frachon, Laurent Guibaud, Cécile Pagan, Magali Pettazzoni, Monique Piraud, Antonin Lamazière, Roseline Froissart

Rapid Communication

Genetic cause and prevalence of hydroxyprolinemia

Christian Staufner, Tobias B. Haack, Patrik Feyh, Gwendolyn Gramer, Deepthi Ediga Raga, Caterina Terrile, Sven Sauer, Jürgen G. Okun, Junmin Fang-Hoffmann, Ertan Mayatepek, Holger Prokisch, Georg F. Hoffmann, Stefan Kölker

Review

Appropriateness of newborn screening for classic galactosaemia: a systematic review

L. Varela-Lema, L. Paz-Valinas, G. Atienza-Merino, R. Zubizarreta-Alberdi, R. Vizoso Villares, M. López-García

Open Access Original Article

Rapid quantification of underivatized amino acids in plasma by hydrophilic interaction liquid chromatography (HILIC) coupled with tandem mass-spectrometry

Hubertus C. M. T. Prinsen, B. G. M. Schiebergen-Bronkhorst, M. W. Roeleveld, J. J. M. Jans, M. G. M. de Sain-van der Velden, G. Visser, P. M. van Hasselt, N. M. Verhoeven-Duif

Original Article

Age at disease onset and peak ammonium level rather than interventional variables predict the neurological outcome in urea cycle disorders

Roland Posset, Angeles Garcia-Cazorla, Vassili Valayannopoulos, Elisa Leão Teles, Carlo Dionisi-Vici, Anaïs Brassier, Alberto B. Burlina, Peter Burgard, Elisenda Cortès-Saladelafont, Dries Dobbelaere, Maria L. Couce, Jolanta Sykut-Cegielska, Johannes Häberle, Allan M. Lund, Anupam Chakrapani, Manuel Schiff, John H. Walter, Jiri Zeman, Roshni Vara, Stefan Kölker

Original Article

Tyrosinemia type I and not treatment with NTBC causes slower learning and altered behavior in mice

Megan A. Hillgartner, Sarah B. Coker, Ashton E. Koenig, Marissa E. Moore, Elizabeth Barnby, Gordon G. MacGregor

Original Article

Qualitative urinary organic acid analysis: 10 years of quality assurance

Verena Peters, James R. Bonham, Georg F. Hoffmann, Camilla Scott, Claus-Dieter Langhans

Original Article

Expanding the molecular diversity and phenotypic spectrum of glycerol 3-phosphate dehydrogenase 1 deficiency

Carlo Dionisi-Vici, Eyal Shteyer, Marcello Niceta, Cristiano Rizzo, Ben Pode-Shakked, Giovanni Chillemi, Alessandro Bruselles, Michela Semeraro, Ortal Barel, Eran Eyal, Nitzan Kol, Yael Haberman, Avishai Lahad, Francesca Diomedi-Camassei, Dina Marek-Yagel, Gideon Rechavi, Marco Tartaglia, Yair Anikster

Open Access Original Article

Glycogen storage disease type III: diagnosis, genotype, management, clinical course and outcome

Christiaan P. Sentner, Irene J. Hoogeveen, David A. Weinstein, René Santer, Elaine Murphy, Patrick J. McKiernan, Ulrike Steuerwald, Nicholas J. Beauchamp, Joanna Taybert, Pascal Laforêt, François M. Petit, Aurélie Hubert, Philippe Labrune, G. Peter A. Smit, Terry G. J. Derks

Open Access Original Article

International Paediatric Mitochondrial Disease Scale

Saskia Koene, Jan C. M. Hendriks, Ilse Dirks, Lonneke de Boer, Maaike C. de Vries, Mirian C. H. Janssen, Izelle Smuts, Cheuk-Wing Fung, Virginia C. N. Wong, I. René F. M. de Coo, Katharina Vill, Claudia Stendel, Thomas Klopstock, Marni J. Falk, Elizabeth M. McCormick, Robert McFarland, Imelda J. M. de Groot, Jan A. M. Smeitink

Original Article

ALG6-CDG: a recognizable phenotype with epilepsy, proximal muscle weakness, ataxia and behavioral and limb anomalies

Eva Morava, Vera Tiemes, Christian Thiel, Nathalie Seta, Pascale de Lonlay, Hans de Klerk, Margot Mulder, Estela Rubio-Gozalbo, Gepke Visser, Peter van Hasselt, Dafne D. G. Horovitz, Carolina Fischinger Moura de Souza, Ida V. D. Schwartz, Andrew Green, Mohammed Al-Owain, Graciella Uziel, Sabine Sigaudy, Brigitte Chabrol, Franc-Jan van Spronsen, Martin Steinert, Eleni Komini, Donald Wurm, Andrea Bevot, Addelkarim Ayadi, Karin Huijben, Marli Dercksen, Peter Witters, Jaak Jaeken, Gert Matthijs, Dirk J. Lefeber, Ron A. Wevers

Original Article

A novel method for determining peroxisomal fatty acid β-oxidation

Masashi Morita, Shun Matsumoto, Airi Okazaki, Kaito Tomita, Shiro Watanabe, Kosuke Kawaguchi, Daishiro Minato, Yuji Matsuya, Nobuyuki Shimozawa, Tsuneo Imanaka

Original Article

The value of plasma vitamin B6 profiles in early onset epileptic encephalopathies

Déborah Mathis, Lucia Abela, Monique Albersen, Céline Bürer, Lisa Crowther, Karin Beese, Hans Hartmann, Levinus A. Bok, Eduard Struys, Sorina M. Papuc, Anita Rauch, Martin Hersberger, Nanda M. Verhoeven-Duif, Barbara Plecko

Original Article

Phenotyping GABA transaminase deficiency: a case description and literature review

Pedro Louro, Lina Ramos, Conceição Robalo, Cândida Cancelinha, Alexandra Dinis, Ricardo Veiga, Raquel Pina, Olinda Rebelo, Ana Pop, Luísa Diogo, Gajja S. Salomons, Paula Garcia

Erratum

Erratum to: Untargeted metabolomic analysis for the clinical screening of inborn errors of metabolism

Marcus J. Miller, Adam D. Kennedy, Andrea D. Eckhart, Lindsay C. Burrage, Jacob E. Wulff, Luke A. D. Miller, Michael V. Milburn, John A. Ryals, Arthur L. Beaudet, Qin Sun, V. Reid Sutton, Sarah H. Elsea

Erratum

Erratum to: ALG6-CDG: a recognizable phenotype with epilepsy, proximal muscle weakness, ataxia and behavioral and limb anomalies

Eva Morava, Vera Tiemes, Christian Thiel, Nathalie Seta, Pascale de Lonlay, Hans de Klerk, Margot Mulder, Estela Rubio-Gozalbo, Gepke Visser, Peter van Hasselt, Dafne D. G. Horovitz, Carolina Fischinger Moura de Souza, Ida V. D. Schwartz, Andrew Green, Mohammed Al-Owain, Graciella Uziel, Sabine Sigaudy, Brigitte Chabrol, Franc-Jan van Spronsen, Martin Steinert, Eleni Komini, Donald Wurm, Andrea Bevot, Addelkarim Ayadi, Karin Huijben, Marli Dercksen, Peter Witters, Jaak Jaeken, Gert Matthijs, Dirk J. Lefeber, Ron A. Wevers

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