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1 . Academic Journal
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
저자
by
Castaman, Giancarlo
;
Crary, Shelley E
;
Escobar, Miguel
;
Gomez, Esteban
;
Haley, Kristina M
, et al.
소스
In
The Lancet Haematology
April 2024 11(4):e265-e275
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7 . Academic Journal
Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 studies
저자
by
Mahlangu, Johnny
;
Jiménez-Yuste, Víctor
;
Ventriglia, Giuliana
;
Niggli, Markus
;
Barlera, Simona
, et al.
소스
In
Research and Practice in Thrombosis and Haemostasis
February 2024 8(2)
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7 . Academic Journal
Pharmacokinetics and coagulation biomarkers in children and adults with hemophilia A receiving emicizumab prophylaxis every 1, 2, or 4 weeks
저자
by
Kiialainen, Anna
;
Adamkewicz, Joanne I.
;
Petry, Claire
;
Oldenburg, Johannes
;
Pipe, Steven W.
, et al.
소스
In
Research and Practice in Thrombosis and Haemostasis
January 2024 8(1)
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7 . Academic Journal
Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline
저자
by
Di Minno, Giovanni
;
Spadarella, Gaia
;
Maldonato, Nelson Mauro
;
De Lucia, Natascia
;
Castaman, Giancarlo
, et al.
소스
In
Blood Reviews
November 2023 62
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7 . Academic Journal
Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy
저자
by
von Drygalski, Annette
;
Gomez, Esteban
;
Giermasz, Adam
;
Castaman, Giancarlo
;
Key, Nigel S.
, et al.
소스
In
Blood Advances
10 October 2023 7(19):5671-5679
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7 . Academic Journal
von Willebrand factor neutralizing and non-neutralizing alloantibodies in 213 subjects with type 3 von Willebrand disease enrolled in 3WINTERS-IPS
저자
by
Pagliari, Maria Teresa
;
Budde, Ulrich
;
Baronciani, Luciano
;
Eshghi, Peyman
;
Ahmadinejad, Minoo
, et al.
소스
In
Journal of Thrombosis and Haemostasis
April 2023 21(4):787-799
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7 . Academic Journal
The p.P1127S pathogenic variant lowers von Willebrand factor levels through higher affinity for the macrophagic scavenger receptor LRP1: Clinical phenotype and pathogenic mechanisms
저자
by
Sacco, Monica
;
Lancellotti, Stefano
;
Branchini, Alessio
;
Tardugno, Maira
;
Testa, Maria Francesca
, et al.
소스
In
Journal of Thrombosis and Haemostasis
August 2022 20(8):1818-1829
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7 . Academic Journal
The bleeding phenotype in people with nonsevere hemophilia
저자
by
Kloosterman, Fabienne R.
;
Zwagemaker, Anne-Fleur
;
Bagot, Catherine N.
;
Beckers, Erik A.M.
;
Castaman, Giancarlo
, et al.
소스
In
Blood Advances
26 July 2022 6(14):4256-4265
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7 . Academic Journal
Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results
저자
by
Leebeek, Frank W.G.
;
Peyvandi, Flora
;
Escobar, Miguel
;
Tiede, Andreas
;
Castaman, Giancarlo
, et al.
소스
In
Blood
14 July 2022 140(2):89-98
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7 . Academic Journal
Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors
저자
by
Shapiro, Amy D.
;
Angchaisuksiri, Pantep
;
Astermark, Jan
;
Benson, Gary
;
Castaman, Giancarlo
, et al.
소스
In
Blood Advances
14 June 2022 6(11):3422-3432
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