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  • Clinical trials for neurodevelopmental disorders: At a therapeutic frontier | Science Translational Medicine
    You are currently viewing the abstract View Full Text Username Enter your Sciencemag org username Password Enter the password that accompanies your username Forgot your username or password Log in Join Subscribe Purchase Article Activate Member Account Renew Subscription Recommend a subscription to your library Help for librarians Abstract A well powered clinical trial that failed to replicate promising results in animal models of fragile X syndrome yields important lessons for clinical trial design Berry Kravis et al this issue Copyright 2016 American Association for the Advancement of Science View Full Text Science Translational Medicine Vol 8 Issue 321 13 January 2016 Table of Contents Article Tools Email Thank you for your interest in spreading the word about Science Translational Medicine NOTE We only request your email address so that the person you are recommending the page to knows that you wanted them to see it and that it is not junk mail We do not capture any email address Your Email Your Name Send To Enter multiple addresses on separate lines or separate them with commas You are going to email the following Clinical trials for neurodevelopmental disorders At a therapeutic frontier Message Subject Your Name has forwarded a page to you from Science Translational Medicine Message Body Your Name thought you would like to see this page from the Science Translational Medicine web site Your Personal Message Send Message Download Powerpoint Print Save to my folders User Name Password Remember my user name password Submit Alerts Please log in to add an alert for this article Username Enter your Sciencemag org username Password Enter the password that accompanies your username Log in Request Permissions Citation tools Clinical trials for neurodevelopmental disorders At a therapeutic frontier By Shafali S Jeste Daniel H Geschwind Science Translational Medicine 13 Jan 2016

    Original URL path: http://stm.sciencemag.org/content/8/321/321fs1.abstract (2016-02-10)
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  • Personalized medical education: Reappraising clinician-scientist training | Science Translational Medicine
    author on Google Scholar Find this author on PubMed Search for this author on this site Alastair M Buchan Radcliffe Department of Medicine University of Oxford Oxford OX3 9DU UK Medical Sciences Division University of Oxford Oxford OX3 9DU UK Find this author on Google Scholar Find this author on PubMed Search for this author on this site Article Figures Data Info Metrics eLetters PDF You are currently viewing the abstract View Full Text Abstract Revitalizing the Oslerian ideal of the clinician scientist teacher may help in the training of the next generation of translational researchers Copyright 2016 American Association for the Advancement of Science View Full Text Science Translational Medicine Vol 8 Issue 321 13 January 2016 Table of Contents Article Tools Email Thank you for your interest in spreading the word about Science Translational Medicine NOTE We only request your email address so that the person you are recommending the page to knows that you wanted them to see it and that it is not junk mail We do not capture any email address Your Email Your Name Send To Enter multiple addresses on separate lines or separate them with commas You are going to email the following Personalized medical education Reappraising clinician scientist training Message Subject Your Name has forwarded a page to you from Science Translational Medicine Message Body Your Name thought you would like to see this page from the Science Translational Medicine web site Your Personal Message Send Message Download Powerpoint Print Save to my folders User Name Password Remember my user name password Submit Alerts Please log in to add an alert for this article Username Enter your Sciencemag org username Password Enter the password that accompanies your username Log in Request Permissions Citation tools Personalized medical education Reappraising clinician scientist training By

    Original URL path: http://stm.sciencemag.org/content/8/321/321fs2 (2016-02-10)
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  • Personalized medical education: Reappraising clinician-scientist training | Science Translational Medicine
    1960 to 58 in 1961 2015 The shift in predominant career pathways of Nobel Laureates in Physiology or Medicine from clinician scientists to nonclinically trained scientists in part reflects the shift of the most awarded areas from human diseases until 1960 to basic molecular biology and genetics after 1960 The downside of this shift is that most basic science discoveries have yet to be translated into therapies and improved patient care whereas the burden of chronic disease associated with the 21st century s aging population is escalating Download high res image Open in new tab Download Powerpoint Fig 1 The greatest prize of all Shown are Nobel Laureates in Physiology or Medicine by medical degree 1901 2015 A steady decline in the proportion of clinician scientists awarded the Nobel Prize in Physiology or Medicine has led to a shift in predominant career pathways of Nobel Laureates in Physiology or Medicine from clinician scientists to nonclinically trained scientists Bars indicate proportions figures in the bars indicate absolute numbers A Nobel Prize was not awarded in 1915 1918 1921 1925 or 1940 1942 Source Biographies of Nobel Laureates on the Nobel Prize website 4 CREDIT C BICKEL SCIENCE TRANSLATIONAL MEDICINE INTEGRATION OF CLINICAL AND RESEARCH EDUCATION After a crisis in the numbers of clinician scientists in the UK in the early 2000s government industry medical research charities and universities recognized the need to strengthen clinical research and innovation in the National Health Service NHS for the benefit of patients To achieve this a wide group of stakeholders chaired by Mark Walport then director of the Wellcome Trust set out to modernize medical careers in the UK 5 and the government created the National Institute for Health Research NIHR 6 Within this framework Oxford academic and healthcare partners established the Oxford University Clinical Academic Graduate School OUCAGS 7 which is aimed at better integrating the exposure to clinical practice research and teaching as advocated by Osler In addition to traditional masters and doctoral programs for aspiring clinician scientists OUCAGS offers new NIHR funded training pathways based on Walport s recommendations academic clinical fellowships allowing trainees to undertake 25 research and 75 clinical training over 3 to 4 years usually to develop a proposal for a doctorate and clinical lectureships allowing trainees to undertake 50 research and 50 clinical training over 4 years usually to conduct postdoctoral research and teaching The faculty and trainees are particularly enthusiastic about these new pathways because they attempt to bridge the organizational divide between the university and the hospital and enable trainees to develop and pursue their own line of research Moreover funding for academic clinical fellowship and clinical lectureship posts comes predominantly from NIHR reflecting the societal importance of translational clinical research Although the implementation of NIHR and OUCAGS has helped to narrow the gap between clinician and scientist it has highlighted the pressing need to develop more integrated dynamic and flexible clinician scientist training programs that span the entire continuum of undergraduate and postgraduate education and training We believe that to achieve this we need to revitalize the Oslerian ideal of the clinician scientist teacher First the integration of clinical training and exposure to research needs to be strengthened Second teaching and mentorship need to be equal partners with research and patient care Third scientifically innovative translational research for patient benefit needs to become the raison d être of clinician scientist training and careers It is time for a seismic shift in the way the next generation of clinician scientists is educated Exposure to and involvement in clinical translational research throughout medical school and postgraduate specialty training is a key tenet Trainees interested in a clinician scientist career should be given an opportunity to undertake a simultaneous parallel research path during medical school and residency Protected time would be given to pursue short research projects relevant to curricular modules of interest throughout the medical school course For example a student learning the fundamentals of cardiology in the classroom or at the bedside could take a short detour from their standard medical course to pursue a research project in that field The duration of dedicated research project time would need to be flexible such as 2 to 6 months to accommodate project goals and demands This would facilitate the introduction and consolidation of core medical principles while contributing to cutting edge science Trainees would be expected to devise a personalized curriculum to enable them to weave research in and out of the regular medical school and residency curriculum In so doing contextual knowledge and real world experiences clinical and scientific would be married through a problem solving based approach Time allotted to the acquisition of core medical knowledge bedside apprenticeship and research training would be dependent on competency based outcomes and not restricted to time fixed rotations The duration of research exposure throughout the degree course would vary according to the trainees needs and interests with the candidate potentially being awarded a higher research degree such as MSc or PhD arising from their scientific contributions This system would provide a distinct platform to arm the emerging clinician scientist with the skills needed to be adequately trained in providing an excellent clinical service at the bedside while taking scientific risks at the bench for the betterment of patient care VALUE OF TEACHING AND MENTORSHIP Critical to the success of such a system would be close mentorship of trainees by dedicated established clinician scientists similar to the Oslerian ideal The personalized nature of the proposed training program would require the close guidance and regular critical appraisal of a trainee s performance from an assigned mentor who would oversee their clinical and research activities Enthusiastic clinical researchers from a range of specialties would supervise research projects while interacting regularly with the trainee s mentor to discuss progress and to mobilize support The time intensive nature of such an apprenticeship would necessarily limit the number of trainees selected on a competitive basis to pursue this path less than 10 of a

    Original URL path: http://stm.sciencemag.org/content/8/321/321fs2.full (2016-02-10)
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  • Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials | Science Translational Medicine
    Cytogénétique Unité Fonctionnelle de Génétique Médicale Assistance Publique Hôpitaux de Paris Groupe Hospitalier Pitié Salpêtrière 75013 Paris France Find this author on Google Scholar Find this author on PubMed Search for this author on this site Jeannie Visootsak Department of Human Genetics Emory University School of Medicine Atlanta GA 30322 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Marc Brinkman Novartis Pharma GmbH 90429 Nürnberg Germany Find this author on Google Scholar Find this author on PubMed Search for this author on this site Karin Rerat Novartis Pharmaceuticals SAS 92500 Rueil Malmaison France Find this author on Google Scholar Find this author on PubMed Search for this author on this site Barbara Koumaras Neurodegeneration Global Development Novartis Pharmaceuticals Corporation East Hanover NJ 07936 1080 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Liansheng Zhu Integrated Quantitative Science Global Development Novartis Pharmaceuticals Corporation East Hanover NJ 07936 1080 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Gottfried Maria Barth Department of Child and Adolescent Psychiatry University Hospital of Tübingen 72076 Tübingen Baden Württemberg Germany Find this author on Google Scholar Find this author on PubMed Search for this author on this site Thomas Jaecklin Neuroscience Development Novartis Pharma AG CH 4056 Basel Switzerland Find this author on Google Scholar Find this author on PubMed Search for this author on this site George Apostol Neuroscience Development Novartis Pharma AG CH 4056 Basel Switzerland Find this author on Google Scholar Find this author on PubMed Search for this author on this site Florian von Raison Neuroscience Development Novartis Pharma AG CH 4056 Basel Switzerland Find this author on Google Scholar Find this author on PubMed Search for this author on this site Article Figures Data Info Metrics eLetters PDF You are currently viewing the editor s summary View Full Text Username Enter your Sciencemag org username Password Enter the password that accompanies your username Forgot your username or password Log in Join Subscribe Purchase Article Activate Member Account Renew Subscription Recommend a subscription to your library Help for librarians The mGluR theory of fragile X put to the test People with the genetic disorder fragile X syndrome exhibit a variable constellation of debilitating physical and cognitive problems Promising evidence from mouse models had raised hopes that an overactive glutamate signaling pathway mGluR was a smoking gun at the heart of the disease and that it could be successfully repaired A pilot study in patients supported the mouse work Down regulation of mGluR improved behavioral problems at least in patients carrying a certain genetic methylation marker Here in a larger well powered clinical trial these results are put to the test and come up short In adolescent or adult fragile X patients whether they have the methylation marker or not the glutamate antagonist mavoglurant had no effect on patient behavior The

    Original URL path: http://stm.sciencemag.org/content/8/321/321ra5.editor-summary (2016-02-10)
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  • Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials | Science Translational Medicine
    Center Sacramento CA 95817 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Sébastien Jacquemont Centre Hospitalier Universitaire Vaudois CH 1011 Lausanne Switzerland CHU Sainte Justine Research Centre Montreal Quebec H3T 1C5 Canada Find this author on Google Scholar Find this author on PubMed Search for this author on this site Perrine Charles Département de Génétique et Cytogénétique Unité Fonctionnelle de Génétique Médicale Assistance Publique Hôpitaux de Paris Groupe Hospitalier Pitié Salpêtrière 75013 Paris France Find this author on Google Scholar Find this author on PubMed Search for this author on this site Jeannie Visootsak Department of Human Genetics Emory University School of Medicine Atlanta GA 30322 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Marc Brinkman Novartis Pharma GmbH 90429 Nürnberg Germany Find this author on Google Scholar Find this author on PubMed Search for this author on this site Karin Rerat Novartis Pharmaceuticals SAS 92500 Rueil Malmaison France Find this author on Google Scholar Find this author on PubMed Search for this author on this site Barbara Koumaras Neurodegeneration Global Development Novartis Pharmaceuticals Corporation East Hanover NJ 07936 1080 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Liansheng Zhu Integrated Quantitative Science Global Development Novartis Pharmaceuticals Corporation East Hanover NJ 07936 1080 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Gottfried Maria Barth Department of Child and Adolescent Psychiatry University Hospital of Tübingen 72076 Tübingen Baden Württemberg Germany Find this author on Google Scholar Find this author on PubMed Search for this author on this site Thomas Jaecklin Neuroscience Development Novartis Pharma AG CH 4056 Basel Switzerland Find this author on Google Scholar Find this author on PubMed Search for this author on this site George Apostol Neuroscience Development Novartis Pharma AG CH 4056 Basel Switzerland Find this author on Google Scholar Find this author on PubMed Search for this author on this site Florian von Raison Neuroscience Development Novartis Pharma AG CH 4056 Basel Switzerland Find this author on Google Scholar Find this author on PubMed Search for this author on this site Article Figures Data Info Metrics eLetters PDF Log in to view full text Username Enter your Sciencemag org username Password Enter the password that accompanies your username Forgot your username or password Log in Join Subscribe Purchase Article Activate Member Account Renew Subscription Recommend a subscription to your library Help for librarians Science Translational Medicine Vol 8 Issue 321 13 January 2016 Table of Contents Article Tools Email Thank you for your interest in spreading the word about Science Translational Medicine NOTE We only request your email address so that the person you are recommending the page to knows that you wanted them to see it and that it is not junk mail We do not capture any email address Your

    Original URL path: http://stm.sciencemag.org/content/8/321/321ra5.full (2016-02-10)
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  • Repetitive blast exposure in mice and combat veterans causes persistent cerebellar dysfunction | Science Translational Medicine
    Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Kole D Meeker Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Brian C Kraemer Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Department of Medicine University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Eric C Petrie Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Murray A Raskind Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Elaine R Peskind Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site David G Cook Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Department of Medicine University of Washington Seattle WA 98195 USA Department of Pharmacology University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Article Figures Data Info Metrics eLetters PDF You are currently viewing the editor s summary View Full Text Username Enter your Sciencemag org username Password Enter the password that accompanies your username Forgot your username or password Log in Join Subscribe Purchase Article Activate Member Account Renew Subscription Recommend a subscription to your library Help for librarians The cerebellum is vulnerable to blast injury in mice and combat veterans Mild traumatic brain injury TBI is often referred to as the signature injury of the wars in Iraq and Afghanistan Most of these TBIs are blast related Currently there is limited understanding of how mild blast causes persistent brain injuries There is also limited insight into how blast induced brain injuries in animal models correspond to humans with mild TBI Meabon et

    Original URL path: http://stm.sciencemag.org/content/8/321/321ra6.editor-summary (2016-02-10)
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  • Repetitive blast exposure in mice and combat veterans causes persistent cerebellar dysfunction | Science Translational Medicine
    Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Ge Li Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Kole D Meeker Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Brian C Kraemer Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Department of Medicine University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Eric C Petrie Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Murray A Raskind Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Elaine R Peskind Mental Illness Research Education and Clinical Center VA Puget Sound Health Care System VA Puget Sound Seattle WA 98108 USA Department of Psychiatry and Behavioral Sciences University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site David G Cook Geriatric Research Education and Clinical Center VA Puget Sound Health Care System Seattle WA 98108 USA Department of Medicine University of Washington Seattle WA 98195 USA Department of Pharmacology University of Washington Seattle WA 98195 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Article Figures Data Info Metrics eLetters PDF Log in to view full text Username Enter your Sciencemag org username Password Enter the password that accompanies your username Forgot your username or password Log in Join Subscribe Purchase Article Activate Member Account Renew Subscription Recommend a subscription to your library Help for librarians Science Translational Medicine Vol 8 Issue 321 13 January 2016 Table of Contents Article Tools Email Thank you for your interest in spreading the word about Science Translational Medicine NOTE We

    Original URL path: http://stm.sciencemag.org/content/8/321/321ra6.full (2016-02-10)
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  • Inhibition of diacylglycerol kinase α restores restimulation-induced cell death and reduces immunopathology in XLP-1 | Science Translational Medicine
    on Google Scholar Find this author on PubMed Search for this author on this site Senta M Kapnick Genetic Disease Research Branch National Human Genome Research Institute National Institutes of Health Bethesda MD 20892 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Katherine Verbist Department of Oncology St Jude Children s Research Hospital Memphis TN 38105 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Paige Tedrick Department of Oncology St Jude Children s Research Hospital Memphis TN 38105 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Pamela L Schwartzberg Genetic Disease Research Branch National Human Genome Research Institute National Institutes of Health Bethesda MD 20892 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Cosima T Baldari Department of Life Sciences University of Siena 53100 Siena Italy Find this author on Google Scholar Find this author on PubMed Search for this author on this site Ignacio Rubio Integrated Research and Treatment Center Center for Sepsis Control and Care and Institute of Molecular Cell Biology Center for Molecular Biomedicine Jena University Hospital D 07745 Jena Germany Find this author on Google Scholar Find this author on PubMed Search for this author on this site Kim E Nichols Department of Oncology St Jude Children s Research Hospital Memphis TN 38105 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Andrew L Snow Department of Pharmacology and Molecular Therapeutics Uniformed Services University of the Health Sciences Bethesda MD 20814 USA Find this author on Google Scholar Find this author on PubMed Search for this author on this site Gianluca Baldanzi Department of Translational Medicine and Institute for Research and Cure of Autoimmune Diseases University of Piemonte Orientale 28100 Novara Italy Find this author on Google Scholar Find this author on PubMed Search for this author on this site Andrea Graziani Department of Translational Medicine and Institute for Research and Cure of Autoimmune Diseases University of Piemonte Orientale 28100 Novara Italy School of Medicine University Vita e Salute San Raffaele 20132 Milan Italy Find this author on Google Scholar Find this author on PubMed Search for this author on this site Article Figures Data Info Metrics eLetters PDF You are currently viewing the editor s summary View Full Text Username Enter your Sciencemag org username Password Enter the password that accompanies your username Forgot your username or password Log in Join Subscribe Purchase Article Activate Member Account Renew Subscription Recommend a subscription to your library Help for librarians SAPping immunopathology Individuals with deficient immune systems may also paradoxically experience hyperimmune side effects X linked lymphoproliferative disease XLP 1 an immunodeficiency caused by defects in the T cell receptor adaptor protein SAP signaling lymphocytic activation molecule SLAM associated protein is associated with

    Original URL path: http://stm.sciencemag.org/content/8/321/321ra7.editor-summary (2016-02-10)
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