Intravenous ferric derisomaltose in patients with heart failure and iron deficiency in the UK (IRONMAN): an investigator-initiated, prospective, randomised, open-label, blinded-endpoint trial

Autor: Paul R Kalra, John G F Cleland, Mark C Petrie, Elizabeth A Thomson, Philip A Kalra, Iain B Squire, Fozia Z Ahmed, Abdallah Al-Mohammad, Peter J Cowburn, Paul W X Foley, Fraser J Graham, Alan G Japp, Rebecca E Lane, Ninian N Lang, Andrew J Ludman, Iain C Macdougall, Pierpaolo Pellicori, Robin Ray, Michele Robertson, Alison Seed, Ian Ford, John GF Cleland, Paul WX Foley, Nicholas Boon, Shannon Amoils, Callum Chapman, Thomas G Diness, John McMurray, Richard Mindham, Pamela Sandu, Claes C Strom, Maureen Travers, Robert Wilcox, Allan Struthers, Patrick Mark, Christopher Weir, Elena Cowan, Charlotte Turner, Rosalynn Austin, Paula Rogers, Badri Chandrasekaran, Eva Fraile, Lynsey Kyeremeh, Lorraine McGregor, Joanna Osmanska, Barbara Meyer, Faheem Ahmad, Jude Fisher, Christina Summersgill, Katarzyna Adeniji, Rajkumar Chinnadurai, Lisa Massimo, Clare Hardman, Daisy Sykes, Sarah Frank, Simon Smith, Mohamed Anwar, Beth Whittington, Vennessa Sookhoo, Sinead Lyons, Janet Middle, Kay Housley, Andrew Clark, Jeanne Bulemfu, Christopher Critoph, Victor Chong, Stephen Wood, Benjamin Szwejkowski, Chim Lang, Jackie Duff, Susan MacDonald, Rebekah Schiff, Patrick Donnelly, Thuraia Nageh, Swapna Kunhunny, Roy Gardner, Marion McAdam, Elizabeth McPherson, Prithwish Banerjee, Eleanor Sear, Nigel Edwards, Jason Glover, Clare Murphy, Justin Cooke, Charles Spencer, Mark Francis, Iain Matthews, Hayley McKie, Andrew Marshall, Janet Large, Jenny Stratford, Piers Clifford, Christopher Boos, Philip Keeling, Debbie Hughes, Aaron Wong, Deborah Jones, Alex James, Rhys Williams, Stephen Leslie, Jim Finlayson, Andrew Hannah, Philip Campbell, John Walsh, Jane Quinn, Susan Piper, Sheetal Patale, Preeti Gupta, Victor Sim, Lucy Knibbs, Kristopher Lyons, Lana Dixon, Colin Petrie, Yuk-ki Wong, Catherine Labinjoh, Simon Duckett, Ian Massey, Henry Savage, Sofia Matias, Jonaifah Ramirez, Charlotte Manisty, Ifza Hussain, Rajiv Sankaranarayanan, Gershan Davis, Samuel McClure, John Baxter, Eleanor Wicks, Jolanta Sobolewska, Jerry Murphy, Ahmed Elzayat, Alastair Cooke, Jay Wright, Simon Williams, Amal Muthumala, Parminder Chaggar, Sue Webber, Gethin Ellis, Mandie Welch, Sudantha Bulugahapitiya, Thomas Jackson, Tapesh Pakrashi, Ameet Bakhai, Vinodh Krishnamurthy, Reto Gamma, Susan Ellery, Geraint Jenkins, Gladdys Thomas, Angus Nightingale, Nicola Greenlaw, Kirsty Wetherall, Ross Clarke, Christopher Graham, Sharon Kean, Alan Stevenson, Robbie Wilson, Sarah Boyle, John McHugh, Lisa Hall, Joanne Woollard, Claire Brunton, Eleanor Dinnett, Amanda Reid, Serena Howe, Jill Nicholls, Anna Cunnington, Elizabeth Douglas, Margaret Fegen, Marc Jones, Sheila McGowan, Barbara Ross, Pamela Surtees, Debra Stuart

Publikováno v: The Lancet. 400:2199-2209

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::d9d13332efd162c59d06dddbfdb09ef8
https://doi.org/10.1016/s0140-6736(22)02083-9

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Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes

Autor: Raghuraman C. Srinivasan, Carl Jorns, Anna Forslöw, Johannes Häberle, Burcu Bestas, Tanja Scherer, Mike Firth, Towe Jakobsson, Barry Rosen, Gabriella Allegri, Olav Rooyackers, Susanna Engberg, Christina Hammarstedt, Beat Thöny, Pinar Akcakaya, Marcello Maresca, Natalie Van Zuydam, Ewa Ellis, Nicole Rimann, Mihaela Zabulica, Stephen C. Strom, Tomas Jakobsson, Roberto Gramignoli, Georgios Makris

Publikováno v: Mol Ther

Ornithine transcarbamylase deficiency (OTCD) is a monogenic disease of ammonia metabolism in hepatocytes. Severe disease is frequently treated by orthotopic liver transplantation. An attractive approach is the correction of a patient's own cells to r

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a6f7a6b2fa12b8edad953796c2d8937b
https://doi.org/10.1016/j.ymthe.2021.01.024

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