檢索結果 - Sarah Mangles

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  1. 1
    Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

    Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer 由 Amit C. Nathwani, Ulrike M. Reiss, Edward G. D. Tuddenham, Pratima Chowdary, Jenny McIntosh, Anne Riddell, Jun Pie, Johnny Mahlangu, Michael Recht, Yu‐Min Shen, Kathleen G. Halka, Michael M. Meagher, Arthur W. Nienhuis, Andrew M. Davidoff, Sarah Mangles, Chris L Morton, Junfang Zhou, Vlad C. Radulescu

    出版 2018
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  2. 2
    The Hemophilia Joint Health Score version 2.1 Validation in Adult Patients Study: A multicenter international study

    The Hemophilia Joint Health Score version 2.1 Validation in Adult Patients Study: A multicenter international study 由 Jean St‐Louis, Audrey Abad, Sharon Funk, Merlyn Tilak, Stephen Classey, N. Zourikian, Paul McLaughlin, Sébastien Lobet, Grace Hernandez, Stacie Akins, Anna J. Wells, Marilyn J. Manco‐Johnson, Judy Ann John, Steve Austin, Pratima Chowdary, Cédric Hermans, Diane J. Nugent, Nihal Bakeer, Sarah Mangles, Pamela Hilliard, Victor S. Blanchette, Brian M. Feldman

    出版 2022
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  3. 3
    Germline mutations in the transcription factor IKZF5 cause thrombocytopenia

    Germline mutations in the transcription factor IKZF5 cause thrombocytopenia 由 Claire Lentaigne, Daniel Greene, Suthesh Sivapalaratnam, Rémi Favier, Denis Seyres, Chantal Thys, Luigi Grassi, Sarah Mangles, Keith Sibson, Matthew Stubbs, Frances Burden, Jean‐Claude Bordet, Corinne Armari‐Alla, Wendy N. Erber, Samantha Farrow, Nicholas Gleadall, Keith Gomez, Karyn Mégy, Sofia Papadia, Christopher J. Penkett, Matthew C. Sims, Luca Stefanucci, Jonathan Stephens, Randy J. Read, Kathleen Stirrups, Willem H. Ouwehand, Michael Laffan, Mattia Frontini, Kathleen Freson, Ernest Turro

    出版 2019
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    De Novo Truncating Mutations in WASF1 Cause Intellectual Disability with Seizures

    De Novo Truncating Mutations in WASF1 Cause Intellectual Disability with Seizures 由 Yoko Itō, Keren Carss, Sofia Duarte, Taila Hartley, Boris Keren, Manju A. Kurian, Isabelle Marey, Perinne Charles, Carla Mendonça, Caroline Nava, Rolph Pfundt, Alba Sanchis‐Juan, Hans van Bokhoven, Anthony van Essen, Conny M.A. van Ravenswaaij‐Arts, Kym M. Boycott, Kristin D. Kernohan, Sarah Dyack, F. Lucy Raymond, Timothy J. Aitman, David Bennett, Mark J. Caulfield, Patrick F. Chinnery, Daniel P. Gale, Ania Koziell, Taco W. Kuijpers, Michael Laffan, Eamonn R. Maher, Hugh S. Markus, Nicholas W. Morrell, Willem H. Ouwehand, David J. Perry, F. Lucy Raymond, Irene Roberts, Kenneth G. C. Smith, Adrian J. Thrasher, Hugh Watkins, Catherine Williamson, Geoffrey Woods, Sofie Ashford, John R. Bradley, Debra Fletcher, Tracey Hammerton, Roger James, Nathalie Kingston, Christopher J. Penkett, Kathleen Stirrups, Marijke Veltman, Tim Young, Matthew A. Brown, Emma Clement, John Davis, Eleanor Dewhurst, Helen Dolling, Marie Erwood, Amy Frary, Rachel Linger, Jennifer M. Martin, Sofia Papadia, Karola Rehnström, Hannah Stark, David Allsup, Steve Austin, Tamam Bakchoul, Tadbir K. Bariana, Paula Bolton‐Maggs, Elizabeth Chalmers, Janine Collins, Peter Collins, Wendy N. Erber, Tamara Everington, Rémi Favier, Kathleen Freson, Bruce Furie, Michael Gattens, Johanna Gebhart, Keith Gomez, Daniel Greene, Andreas Greinacher, Paolo Gresele, Daniel Hart, Johan W. M. Heemskerk, Yvonne Henskens, Rashid Kazmi, David Keeling, Anne M. Kelly, Michele P. Lambert, Claire Lentaigne, Ri Liesner, Μichael Μakris, Sarah Mangles, Mary Mathias, Carolyn M. Millar, Andrew Mumford, Paquita Nurden, Jeanette Payne, John Pasi, Kathelijne Peerlinck, Shoshana Revel‐Vilk, Michael Richards

    出版 2018
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