Ten Years after the International Committee of Medical Journal Editors' Clinical Trial Registration Initiative, One Quarter of Phase 3 Pediatric Epilepsy Clinical Trials Still Rema... by Anette Lampert, Georg F Hoffmann, Markus Ries

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Maternal vitamin deficiency mimicking multiple acyl-CoA dehydrogenase deficiency on newborn screening by Gwendolyn Gramer, Georg F. Hoffmann, Julia B. Hennermann

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23rd Annual Meeting of the German Society for Newborn Screening (Deutsche Gesellschaft für Neugeborenenscreening, DGNS) by Gwendolyn Gramer, Jürgen G. Okun, Georg F. Hoffmann

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Thirty Years of Orphan Drug Legislation and the Development of Drugs to Treat Rare Seizure Conditions: A Cross Sectional Analysis. by Jan Henje Döring, Anette Lampert, Georg F Hoffmann, Markus Ries

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An Assessment of Publication Status of Pediatric Liver Transplantation Studies. by Thomas Breil, Daniel Wenning, Ulrike Teufel, Georg F Hoffmann, Markus Ries

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Pregnancy, delivery, and postpartum period in infantile liver failure syndrome type 2 due to variants in NBAS by Bianca Peters, Felix Wiemers, Dominic Lenz, Stefan Kölker, Georg F. Hoffmann, Siegmund Köhler, Christian Staufner

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Monitoring of Intraportal Liver Cell Application in Children by Jochen Meyburg M.D., Friederike Hoerster, Jan Schmidt, Johannes Poeschl, Georg F. Hoffmann, Jens-Peter Schenk

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Risk Factors for Childhood Overweight and Obesity in Ukraine and Germany by Vira Yakovenko, Laura Henn, Markus Bettendorf, Natalia Zelinska, Galyna Soloviova, Georg F. Hoffmann, Juergen Grulich-Henn

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High blood pressure, a red flag for the neonatal manifestation of urea cycle disorders by Ulrike Teufel, Peter Burgard, Jochen Meyburg, Martin Lindner, Johannes Poeschl, Peter Ruef, Georg F. Hoffmann, Stefan Kölker

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Generation of an induced pluripotent stem cell line (DHMCi009-A) from an individual with TUBB2A tubulinopathy by Julian Schröter, Hanna Syring, Gudrun Göhring, Stefan Kölker, Thomas Opladen, Georg F. Hoffmann, Steffen Syrbe, Sabine Jung-Klawitter

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Evaluation of Right Ventricular Function in Patients with Propionic Acidemia—A Cross-Sectional Study by Alexander Kovacevic, Sven F. Garbade, Friederike Hörster, Georg F. Hoffmann, Matthias Gorenflo, Derliz Mereles, Stefan Kölker, Christian Staufner

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FDA orphan drug designations for lysosomal storage disorders - a cross-sectional analysis. by Sven F Garbade, Matthias Zielonka, Konstantin Mechler, Stefan Kölker, Georg F Hoffmann, Christian Staufner, Eugen Mengel, Markus Ries

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Generation of an induced pluripotent stem cell line (DHMCi008-A) from an individual with TUBA1A tubulinopathy by Julian Schröter, Hanna Syring, Gudrun Göhring, Stefan Kölker, Thomas Opladen, Georg F. Hoffmann, Steffen Syrbe, Sabine Jung-Klawitter

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Machine Learning Methods Improve Specificity in Newborn Screening for Isovaleric Aciduria by Elaine Zaunseder, Ulrike Mütze, Sven F. Garbade, Saskia Haupt, Patrik Feyh, Georg F. Hoffmann, Vincent Heuveline, Stefan Kölker

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Clinical Research in Vulnerable Populations: Variability and Focus of Institutional Review Boards' Responses. by Bärbel Kästner, Simone Behre, Nadine Lutz, Friederike Bürger, Steffen Luntz, Katrin Hinderhofer, Martin Bendszus, Georg F Hoffmann, Markus Ries

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Generation of an iPSC line from a patient with infantile liver failure syndrome 2 due to mutations in NBAS: DHMCi004-A by Dominic Lenz, Christian Staufner, Selina Wächter, Maike Hagedorn, Juliane Ebersold, Gudrun Göhring, Stefan Kölker, Georg F. Hoffmann, Sabine Jung-Klawitter

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New Cases of Maleylacetoacetate Isomerase Deficiency with Detection by Newborn Screening and Natural History over 32 Years: Experience from a German Newborn Screening Center by Gwendolyn Gramer, Saskia B. Wortmann, Junmin Fang-Hoffmann, Dirk Kohlmüller, Jürgen G. Okun, Holger Prokisch, Thomas Meitinger, Georg F. Hoffmann

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Primary carnitine deficiency – diagnosis after heart transplantation: better late than never! by Sarah C. Grünert, Sara Tucci, Anke Schumann, Meike Schwendt, Gwendolyn Gramer, Georg F. Hoffmann, Michelle Erbel, Brigitte Stiller, Ute Spiekerkoetter

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Generation of an induced pluripotent stem cell (iPSC) line, DHMCi005-A, from a patient with CALFAN syndrome due to mutations in SCYL1 by Dominic Lenz, Christian Staufner, Selina Wächter, Maike Hagedorn, Juliane Ebersold, Gudrun Göhring, Stefan Kölker, Georg F. Hoffmann, Sabine Jung-Klawitter

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Maternal Vitamin B₁₂ Deficiency Detected by Newborn Screening—Evaluation of Causes and Characteristics by Anna T. Reischl-Hajiabadi, Sven F. Garbade, Patrik Feyh, Karl Heinz Weiss, Ulrike Mütze, Stefan Kölker, Georg F. Hoffmann, Gwendolyn Gramer

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