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Sarah Mangles
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Journal Articles

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

Ulrike M. Reiss, Andrew M. Davidoff, Edward G. D. Tuddenham, Pratima Chowdary, Jenny H. McIntosh, Anne Riddell, Arnulfo Pie, Paul Batty, Joanna C. M. Calvert, Sarah Mangles, Johnny Mahlangu, Kristina M. Haley, Michael Recht, Yu-Min Shen, Kathleen G. Halka, Junfang Zhou, Guolian Kang, Victoria Okhomina, Christopher L Morton, Amit C. Nathwani
Journal: Blood
Blood (2023) 142 (Supplement 1): 1056.
https://doi.org/10.1182/blood-2023-186891
Published: 2023
Journal Articles

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Sylvia Von Mackensen, Pratima Chowdary, Sarah Mangles, Qifeng Yu, Baisong Mei, Shauna R. Andersson, Pronabesh Dasmahapatra, MBBS, MPH
Journal: Blood
Blood (2020) 136 (Supplement 1): 23–24.
https://doi.org/10.1182/blood-2020-142266
Published: 2020
Journal Articles

Germline mutations in the transcription factor IKZF5 cause thrombocytopenia

Claire Lentaigne, Daniel Greene, Suthesh Sivapalaratnam, Remi Favier, Denis Seyres, Chantal Thys, Luigi Grassi, Sarah Mangles, Keith Sibson, Matthew Stubbs, Frances Burden, Jean-Claude Bordet, Corinne Armari-Alla, Wendy Erber, Samantha Farrow, Nicholas Gleadall, Keith Gomez, Karyn Megy, Sofia Papadia, Christopher J. Penkett, Matthew C. Sims, Luca Stefanucci, Jonathan C. Stephens, Randy J. Read, Kathleen E. Stirrups, Willem H. Ouwehand, Michael A. Laffan, NIHR BioResource, Mattia Frontini, Kathleen Freson, Ernest Turro
Journal: Blood
Blood (2019) 134 (23): 2070–2081.
https://doi.org/10.1182/blood.2019000782
Published: 2019
Includes: Supplemental data
Journal Articles

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

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 491.
https://doi.org/10.1182/blood-2018-99-118334
Published: 2018