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Page 1
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. Among authors: mcguinn ce. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural).
Shapiro AD, Ragni MV, Borhany M, Abajas YL, Tarantino MD, Holstein K, Croteau SE, Liesner R, Tarango C, Carvalho M, McGuinn C, Funding E, Kempton CL, Bidlingmaier C, Cohen A, Oldenburg J, Kearney S, Knoll C, Kuriakose P, Acharya S, Reiss UM, Kulkarni R, Witkop M, Lethagen S, Donfield S, LeBeau P, Berntorp E, Astermark J. Shapiro AD, et al. Haemophilia. 2021 Jan;27(1):49-59. doi: 10.1111/hae.14139. Epub 2020 Dec 5. Haemophilia. 2021. PMID: 33278853
The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development.
Reipert BM, Gangadharan B, Hofbauer CJ, Berg V, Schweiger H, Bowen J, Blatny J, Fijnvandraat K, Mullins ES, Klintman J, Male C, McGuinn C, Meeks SL, Radulescu VC, Ragni MV, Recht M, Shapiro AD, Staber JM, Yaish HM, Santagostino E, Brown DL. Reipert BM, et al. Blood Adv. 2020 Nov 24;4(22):5785-5796. doi: 10.1182/bloodadvances.2020002731. Blood Adv. 2020. PMID: 33232473 Free PMC article.
Haemophilia: factoring in new therapies.
Fassel H, McGuinn C. Fassel H, et al. Br J Haematol. 2021 Sep;194(5):835-850. doi: 10.1111/bjh.17580. Epub 2021 Jul 28. Br J Haematol. 2021. PMID: 34322873 Review.
Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain.
Berntorp E, LeBeau P, Ragni MV, Borhany M, Abajas YL, Tarantino MD, Holstein K, Croteau SE, Liesner R, Tarango C, Carvalho M, McGuinn C, Funding E, Kempton CL, Bidlingmaier C, Cohen A, Oldenburg J, Kearney S, Knoll C, Kuriakose P, Acharya S, Reiss UM, Kulkarni R, Witkop M, Lethagen S, Krouse R, Shapiro AD, Astermark J. Berntorp E, et al. Haemophilia. 2022 May;28(3):453-461. doi: 10.1111/hae.14525. Epub 2022 Mar 8. Haemophilia. 2022. PMID: 35263495 Free PMC article.
Pilot trial of risk-adapted cyclophosphamide intensity based conditioning and HLA matched sibling and unrelated cord blood stem cell transplantation in newly diagnosed pediatric and adolescent recipients with acquired severe aplastic anemia.
McGuinn C, Geyer MB, Jin Z, Garvin JH, Satwani P, Bradley MB, Bhatia M, George D, Duffy D, Morris E, van de Ven C, Schwartz J, Baxter-Lowe LA, Cairo MS. McGuinn C, et al. Pediatr Blood Cancer. 2014 Jul;61(7):1289-94. doi: 10.1002/pbc.24976. Epub 2014 Feb 12. Pediatr Blood Cancer. 2014. PMID: 24623601 Clinical Trial.
15 results