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1.

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.

Mahlangu, Johnny; Kaczmarek, Radoslaw; von Drygalski, Annette; Shapiro, Susan; Chou, Sheng-Chieh; Ozelo, Margareth C; Kenet, Gili; Peyvandi, Flora; Wang, Michael; Madan, Bella; Key, Nigel S; Laffan, Michael; Dunn, Amy L; Mason, Jane; Quon, Doris V; Symington, Emily; Leavitt, Andrew D; Oldenburg, Johannes; Chambost, Hervé; Reding, Mark T; Jayaram, Kala; Yu, Hua; Mahajan, Reena; Chavele, Konstantia-Maria; Reddy, Divya B; Henshaw, Joshua; Robinson, Tara M; Wong, Wing Yen; Pipe, Steven W.

N Engl J Med ; 388(8): 694-705, 2023 02 23.

Artículo en Inglés | MEDLINE | ID: mdl-36812433

2.

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.

Pipe, Steven W; Leebeek, Frank W G; Recht, Michael; Key, Nigel S; Castaman, Giancarlo; Miesbach, Wolfgang; Lattimore, Susan; Peerlinck, Kathelijne; Van der Valk, Paul; Coppens, Michiel; Kampmann, Peter; Meijer, Karina; O'Connell, Niamh; Pasi, K John; Hart, Daniel P; Kazmi, Rashid; Astermark, Jan; Hermans, Cedric R J R; Klamroth, Robert; Lemons, Richard; Visweshwar, Nathan; von Drygalski, Annette; Young, Guy; Crary, Shelley E; Escobar, Miguel; Gomez, Esteban; Kruse-Jarres, Rebecca; Quon, Doris V; Symington, Emily; Wang, Michael; Wheeler, Allison P; Gut, Robert; Liu, Ying P; Dolmetsch, Ricardo E; Cooper, David L; Li, Yanyan; Goldstein, Brahm; Monahan, Paul E.

N Engl J Med ; 388(8): 706-718, 2023 02 23.

Artículo en Inglés | MEDLINE | ID: mdl-36812434

3.

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial.

Pipe, Steven W; Collins, Peter; Dhalluin, Christophe; Kenet, Gili; Schmitt, Christophe; Buri, Muriel; Jiménez-Yuste, Víctor; Peyvandi, Flora; Young, Guy; Oldenburg, Johannes; Mancuso, Maria Elisa; Kavakli, Kaan; Kiialainen, Anna; Deb, Sonia; Niggli, Markus; Chang, Tiffany; Lehle, Michaela; Fijnvandraat, Karin.

Blood ; 143(14): 1355-1364, 2024 Apr 04.

Artículo en Inglés | MEDLINE | ID: mdl-38127586

4.

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.

Ozelo, Margareth C; Mahlangu, Johnny; Pasi, K John; Giermasz, Adam; Leavitt, Andrew D; Laffan, Michael; Symington, Emily; Quon, Doris V; Wang, Jiaan-Der; Peerlinck, Kathelijne; Pipe, Steven W; Madan, Bella; Key, Nigel S; Pierce, Glenn F; O'Mahony, Brian; Kaczmarek, Radoslaw; Henshaw, Joshua; Lawal, Adebayo; Jayaram, Kala; Huang, Mei; Yang, Xinqun; Wong, Wing Y; Kim, Benjamin.

N Engl J Med ; 386(11): 1013-1025, 2022 03 17.

Artículo en Inglés | MEDLINE | ID: mdl-35294811

5.

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy.

Itzler, Robbin; Buckner, Tyler W; Leebeek, Frank W G; Miller, Joel; Recht, Michael; Drelich, Douglass; Monahan, Paul E; Pipe, Steven W.

Haemophilia ; 30(3): 709-719, 2024 May.

Artículo en Inglés | MEDLINE | ID: mdl-38462823

6.

Macrophage Galactose Lectin Contributes to the Regulation of FVIII (Factor VIII) Clearance in Mice-Brief Report.

Ward, Soracha E; Guest, Thomas; Byrne, Ciara; Lopes, Patricia; O'Sullivan, Jamie M; Doherty, Dearbhla; O'Connell, David; Gutierrez Llaneza, Sara; Chion, Alain; Fazavana, Judicael; Fallon, Padraic G; Preston, Roger J S; Johnsen, Jill M; Pipe, Steven W; Turecek, Peter L; O'Donnell, James S.

Arterioscler Thromb Vasc Biol ; 43(4): 540-546, 2023 04.

Artículo en Inglés | MEDLINE | ID: mdl-36727518

7.

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies.

Callaghan, Michael U; Negrier, Claude; Paz-Priel, Ido; Chang, Tiffany; Chebon, Sammy; Lehle, Michaela; Mahlangu, Johnny; Young, Guy; Kruse-Jarres, Rebecca; Mancuso, Maria Elisa; Niggli, Markus; Howard, Monet; Bienz, Nives Selak; Shima, Midori; Jiménez-Yuste, Victor; Schmitt, Christophe; Asikanius, Elina; Levy, Gallia G; Pipe, Steven W; Oldenburg, Johannes.

Blood ; 137(16): 2231-2242, 2021 04 22.

Artículo en Inglés | MEDLINE | ID: mdl-33512413

8.

The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing.

Mancuso, Maria Elisa; Mahlangu, Johnny N; Pipe, Steven W.

Lancet ; 397(10274): 630-640, 2021 02 13.

Artículo en Inglés | MEDLINE | ID: mdl-33460559

9.

Gene therapy: Practical aspects of implementation.

Pipe, Steven W; Reddy, K Rajender; Chowdary, Pratima.

Haemophilia ; 28 Suppl 4: 44-52, 2022 May.

Artículo en Inglés | MEDLINE | ID: mdl-35521727

10.

Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.

Pipe, Steven W; Hermans, Cédric; Chitlur, Meera; Carcao, Manuel; Castaman, Giancarlo; Davis, Joanna A; Ducore, Jonathan; Dunn, Amy L; Escobar, Miguel; Journeycake, Janna; Khan, Osman; Mahlangu, Johnny; Meeks, Shannon L; Mitha, Ismail Haroon; Négrier, Claude; Nowak-Göttl, Ulrike; Recht, Michael; Chrisentery-Singleton, Tammuella; Stasyshyn, Oleksandra; Vilchevska, Kateryna V; Martinez, Laura Villarreal; Wang, Michael; Windyga, Jerzy; Young, Guy; Alexander, W Allan; Bonzo, Daniel; Macie, Christopher; Mitchell, Ian S; Sauty, Evelyne; Wilkinson, Thomas A; Shapiro, Amy D.

Haemophilia ; 28(4): 548-556, 2022 Jul.

Artículo en Inglés | MEDLINE | ID: mdl-35475308

11.

Pain and functional disability amongst adults with moderate and severe haemophilia from the Irish personalised approach to the treatment of haemophilia (iPATH) study.

Kennedy, Megan; O' Mahony, Brian; Roche, Sheila; McGowan, Mark; Singleton, Evelyn; Ryan, Kevin; O' Connell, Niamh M; Pipe, Steven W; Lavin, Michelle; O' Donnell, James S; Turecek, Peter L; Gormley, John.

Eur J Haematol ; 108(6): 518-527, 2022 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-35258118

12.

Long-Term Effects of Hemophilia B Gene Therapy. Reply.

Pipe, Steven W; Monahan, Paul E.

N Engl J Med ; 388(20): 1918-1919, 2023 May 18.

Artículo en Inglés | MEDLINE | ID: mdl-37195961

13.

New therapies for hemophilia.

Weyand, Angela C; Pipe, Steven W.

Blood ; 133(5): 389-398, 2019 01 31.

Artículo en Inglés | MEDLINE | ID: mdl-30559264

14.

Delivering on the promise of gene therapy for haemophilia.

Pipe, Steven W.

Haemophilia ; 27 Suppl 3: 114-121, 2021 Feb.

Artículo en Inglés | MEDLINE | ID: mdl-32490590

15.

Management of COVID-19-associated coagulopathy in persons with haemophilia.

Pipe, Steven W; Kaczmarek, Radoslaw; Srivastava, Alok; Pierce, Glenn F; Makris, Mike; Hermans, Cedric.

Haemophilia ; 27(1): 41-48, 2021 Jan.

Artículo en Inglés | MEDLINE | ID: mdl-33216448

16.

The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies.

Skinner, Mark W; Négrier, Claude; Paz-Priel, Ido; Chebon, Sammy; Jiménez-Yuste, Victor; Callaghan, Michael U; Lehle, Michaela; Niggli, Markus; Mahlangu, Johnny; Shapiro, Amy; Shima, Midori; Campinha-Bacote, Avrita; Levy, Gallia G; Oldenburg, Johannes; von Mackensen, Sylvia; Pipe, Steven W.

Haemophilia ; 27(5): 854-865, 2021 Sep.

Artículo en Inglés | MEDLINE | ID: mdl-34171159

17.

Novel therapeutics for hemophilia and other bleeding disorders.

Callaghan, Michael U; Sidonio, Robert; Pipe, Steven W.

Blood ; 132(1): 23-30, 2018 07 05.

Artículo en Inglés | MEDLINE | ID: mdl-29769259

18.

Biological mechanisms underlying inter-individual variation in factor VIII clearance in haemophilia.

Turecek, Peter L; Johnsen, Jill M; Pipe, Steven W; O'Donnell, James S.

Haemophilia ; 26(4): 575-583, 2020 Jul.

Artículo en Inglés | MEDLINE | ID: mdl-32596930

19.

HIF1-alpha Regulates Acinar Cell Function and Response to Injury in Mouse Pancreas.

Park, Min-Jung; Iyer, Sapna; Xue, Xiang; Bragazzi Cunha, Juliana; Gu, Shufang; Moons, David; Pipe, Steven W; Williams, John A; Simeone, Diane M; Shah, Yatrik M; Omary, M Bishr.

Gastroenterology ; 154(6): 1630-1634.e3, 2018 05.

Artículo en Inglés | MEDLINE | ID: mdl-29409830

20.

Lifelong gene therapy in dogs with hemophilia A.