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Impact of MEK Inhibition on Childhood RASopathy-Associated Hypertrophic Cardiomyopathy.

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Title: Impact of MEK Inhibition on Childhood RASopathy-Associated Hypertrophic Cardiomyopathy.
Authors: Wolf CM; Department of Congenital Heart Defects and Pediatric Cardiology, German Heart Center Munich, School of Medicine and Health, Technical University of Munich, Munich, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Munich Heart Alliance, Munich, Germany; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart., Zenker M; Institute of Human Genetics, University Hospital Magdeburg, Otto-von-Guericke-University, Magdeburg, Germany., Boleti O; Centre for Inherited Cardiovascular Diseases, Institute of Cardiovascular Science, University College London and Great Ormond Street Hospital, London, United Kingdom., Norrish G; Centre for Inherited Cardiovascular Diseases, Institute of Cardiovascular Science, University College London and Great Ormond Street Hospital, London, United Kingdom., Russell M; University of Michigan, Ann Arbor, Michigan, USA., Meisner JK; University of Michigan, Ann Arbor, Michigan, USA., Peng DM; University of Michigan, Ann Arbor, Michigan, USA., Prendiville T; Children's Health Ireland at Crumlin, Crumlin, Ireland., Kleinmahon J; Ochsner Hospital for Children, Jefferson, Louisiana, USA., Kantor PF; Children's Hospital Los Angeles, Los Angeles, California, USA., Gottlieb Sen D; Johns Hopkins School of Medicine, Baltimore, Maryland, USA., Human DG; British Columbia's Children's Hospital, Vancouver, British Columbia, Canada., Ewert P; Department of Congenital Heart Defects and Pediatric Cardiology, German Heart Center Munich, School of Medicine and Health, Technical University of Munich, Munich, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Munich Heart Alliance, Munich, Germany; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart., Krueger M; Department of Neonatology, Municipal Hospital Munich Schwabing, Munich, Germany., Reber D; Department of Neonatology, Municipal Hospital Munich Schwabing, Munich, Germany., Donner B; Pediatric Cardiology, University Children's Hospital of Basel, University of Basel, Basel, Switzerland., Hart C; Department of Pediatric Cardiology, Pediatric Heart Center, Children's Hospital, University of Bonn, Bonn, Germany., Odri Komazec I; Department of Child and Adolescent Health (Pediatrics III, Pediatric Cardiology), Medical University of Innsbruck, Innsbruck, Austria., Rupp S; Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany., Hahn A; Department of Child Neurology, University of Giessen, Giessen, Germany., Hanser A; Department of Pediatric Cardiology and Intensive Medicine, University Hospital Tübingen, Eberhard-Karls-University, Tübingen, Germany., Hofbeck M; Department of Pediatric Cardiology and Intensive Medicine, University Hospital Tübingen, Eberhard-Karls-University, Tübingen, Germany., Draaisma JMT; Radboud University Medical Center, Radboud Institute for Health Sciences, Amalia Children's Hospital, Department of Pediatrics, Nijmegen, the Netherlands., Udink Ten Cate FEA; Radboud University Medical Center, Radboud Institute for Health Sciences, Amalia Children's Hospital, Department of Pediatrics, Nijmegen, the Netherlands., Mussa A; Department of Public Health and Pediatric Sciences, University of Turin, Turin, Italy., Ferrero GB; Department of Clinical and Biological Sciences, School of Medicine, University of Turin, Turin, Italy., Vaujois L; Centre Mère-Enfant Soleil, Université de Laval, Quebec City, Quebec, Canada., Raboisson MJ; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada., Delrue MA; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada., Marquis C; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada., Théoret Y; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada., Bogarapu S; Children's Hospital of Illinois, University of Illinois College of Medicine, Chicago, Illinois, USA., Dancea A; Montreal Children's Hospital, McGill University Health Center, Montréal, Quebec, Canada., Handrup MM; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart; Center for Rare Diseases, Aarhus University Hospital, Copenhagen, Denmark., Kemna M; Seattle Children's Hospital, Seattle, Washington, USA., Ojala T; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart; New Children's Hospital Pediatric Research Center, Helsinki University Hospital, Helsinki, Finland., Dham N; George Washington University and Children's National Hospital, Washington, District of Columbia, USA., Dicke F; Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada., Friede T; University Medical Center Göttingen, Department of Medical Statistics, Göttingen, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Lower Saxony, Göttingen, Germany; Deutsches Zentrum für Kinder- und Jugendgesundheit (German Center for Child and Adolescent Health), partner site Göttingen, Göttingen, Germany., Kaski JP; Centre for Inherited Cardiovascular Diseases, Institute of Cardiovascular Science, University College London and Great Ormond Street Hospital, London, United Kingdom., Gelb BD; Mindich Child Health and Development Institute and Departments of Pediatrics and Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA., Andelfinger G; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada. Electronic address: gregor.andelfinger.med@ssss.gouv.qc.ca.
Source: JACC. Basic to translational science [JACC Basic Transl Sci] 2025 Feb; Vol. 10 (2), pp. 152-166. Date of Electronic Publication: 2024 Dec 04.
Publication Type: Journal Article
Journal Info: Publisher: Elsevier on behalf of the American College of Cardiology Foundation Country of Publication: United States NLM ID: 101677259 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2452-302X (Electronic) Linking ISSN: 2452302X NLM ISO Abbreviation: JACC Basic Transl Sci Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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  Data: Impact of MEK Inhibition on Childhood RASopathy-Associated Hypertrophic Cardiomyopathy.
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  Data: <searchLink fieldCode="AU" term="%22Wolf+CM%22">Wolf CM</searchLink>; Department of Congenital Heart Defects and Pediatric Cardiology, German Heart Center Munich, School of Medicine and Health, Technical University of Munich, Munich, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Munich Heart Alliance, Munich, Germany; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart.<br /><searchLink fieldCode="AU" term="%22Zenker+M%22">Zenker M</searchLink>; Institute of Human Genetics, University Hospital Magdeburg, Otto-von-Guericke-University, Magdeburg, Germany.<br /><searchLink fieldCode="AU" term="%22Boleti+O%22">Boleti O</searchLink>; Centre for Inherited Cardiovascular Diseases, Institute of Cardiovascular Science, University College London and Great Ormond Street Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Norrish+G%22">Norrish G</searchLink>; Centre for Inherited Cardiovascular Diseases, Institute of Cardiovascular Science, University College London and Great Ormond Street Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Russell+M%22">Russell M</searchLink>; University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Meisner+JK%22">Meisner JK</searchLink>; University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Peng+DM%22">Peng DM</searchLink>; University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Prendiville+T%22">Prendiville T</searchLink>; Children's Health Ireland at Crumlin, Crumlin, Ireland.<br /><searchLink fieldCode="AU" term="%22Kleinmahon+J%22">Kleinmahon J</searchLink>; Ochsner Hospital for Children, Jefferson, Louisiana, USA.<br /><searchLink fieldCode="AU" term="%22Kantor+PF%22">Kantor PF</searchLink>; Children's Hospital Los Angeles, Los Angeles, California, USA.<br /><searchLink fieldCode="AU" term="%22Gottlieb+Sen+D%22">Gottlieb Sen D</searchLink>; Johns Hopkins School of Medicine, Baltimore, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Human+DG%22">Human DG</searchLink>; British Columbia's Children's Hospital, Vancouver, British Columbia, Canada.<br /><searchLink fieldCode="AU" term="%22Ewert+P%22">Ewert P</searchLink>; Department of Congenital Heart Defects and Pediatric Cardiology, German Heart Center Munich, School of Medicine and Health, Technical University of Munich, Munich, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Munich Heart Alliance, Munich, Germany; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart.<br /><searchLink fieldCode="AU" term="%22Krueger+M%22">Krueger M</searchLink>; Department of Neonatology, Municipal Hospital Munich Schwabing, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Reber+D%22">Reber D</searchLink>; Department of Neonatology, Municipal Hospital Munich Schwabing, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Donner+B%22">Donner B</searchLink>; Pediatric Cardiology, University Children's Hospital of Basel, University of Basel, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Hart+C%22">Hart C</searchLink>; Department of Pediatric Cardiology, Pediatric Heart Center, Children's Hospital, University of Bonn, Bonn, Germany.<br /><searchLink fieldCode="AU" term="%22Odri+Komazec+I%22">Odri Komazec I</searchLink>; Department of Child and Adolescent Health (Pediatrics III, Pediatric Cardiology), Medical University of Innsbruck, Innsbruck, Austria.<br /><searchLink fieldCode="AU" term="%22Rupp+S%22">Rupp S</searchLink>; Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany.<br /><searchLink fieldCode="AU" term="%22Hahn+A%22">Hahn A</searchLink>; Department of Child Neurology, University of Giessen, Giessen, Germany.<br /><searchLink fieldCode="AU" term="%22Hanser+A%22">Hanser A</searchLink>; Department of Pediatric Cardiology and Intensive Medicine, University Hospital Tübingen, Eberhard-Karls-University, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Hofbeck+M%22">Hofbeck M</searchLink>; Department of Pediatric Cardiology and Intensive Medicine, University Hospital Tübingen, Eberhard-Karls-University, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Draaisma+JMT%22">Draaisma JMT</searchLink>; Radboud University Medical Center, Radboud Institute for Health Sciences, Amalia Children's Hospital, Department of Pediatrics, Nijmegen, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Udink+Ten+Cate+FEA%22">Udink Ten Cate FEA</searchLink>; Radboud University Medical Center, Radboud Institute for Health Sciences, Amalia Children's Hospital, Department of Pediatrics, Nijmegen, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Mussa+A%22">Mussa A</searchLink>; Department of Public Health and Pediatric Sciences, University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Ferrero+GB%22">Ferrero GB</searchLink>; Department of Clinical and Biological Sciences, School of Medicine, University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Vaujois+L%22">Vaujois L</searchLink>; Centre Mère-Enfant Soleil, Université de Laval, Quebec City, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Raboisson+MJ%22">Raboisson MJ</searchLink>; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Delrue+MA%22">Delrue MA</searchLink>; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Marquis+C%22">Marquis C</searchLink>; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Théoret+Y%22">Théoret Y</searchLink>; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Bogarapu+S%22">Bogarapu S</searchLink>; Children's Hospital of Illinois, University of Illinois College of Medicine, Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Dancea+A%22">Dancea A</searchLink>; Montreal Children's Hospital, McGill University Health Center, Montréal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Handrup+MM%22">Handrup MM</searchLink>; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart; Center for Rare Diseases, Aarhus University Hospital, Copenhagen, Denmark.<br /><searchLink fieldCode="AU" term="%22Kemna+M%22">Kemna M</searchLink>; Seattle Children's Hospital, Seattle, Washington, USA.<br /><searchLink fieldCode="AU" term="%22Ojala+T%22">Ojala T</searchLink>; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart; New Children's Hospital Pediatric Research Center, Helsinki University Hospital, Helsinki, Finland.<br /><searchLink fieldCode="AU" term="%22Dham+N%22">Dham N</searchLink>; George Washington University and Children's National Hospital, Washington, District of Columbia, USA.<br /><searchLink fieldCode="AU" term="%22Dicke+F%22">Dicke F</searchLink>; Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.<br /><searchLink fieldCode="AU" term="%22Friede+T%22">Friede T</searchLink>; University Medical Center Göttingen, Department of Medical Statistics, Göttingen, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Lower Saxony, Göttingen, Germany; Deutsches Zentrum für Kinder- und Jugendgesundheit (German Center for Child and Adolescent Health), partner site Göttingen, Göttingen, Germany.<br /><searchLink fieldCode="AU" term="%22Kaski+JP%22">Kaski JP</searchLink>; Centre for Inherited Cardiovascular Diseases, Institute of Cardiovascular Science, University College London and Great Ormond Street Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Gelb+BD%22">Gelb BD</searchLink>; Mindich Child Health and Development Institute and Departments of Pediatrics and Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA.<br /><searchLink fieldCode="AU" term="%22Andelfinger+G%22">Andelfinger G</searchLink>; CHU Sainte Justine, Department of Pediatrics, Université de Montréal, Montréal, Quebec, Canada. 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