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Akademický článek

EFFICACY AND SAFETY OF VALOCTOCOGENE ROXAPARVOVEC 4 YEARS AFTER GENE TRANSFER IN GENER8-1

Autor: MC Ozelo, AD Leavitt, J Mahlangu, G Kenet, SW Pipe, S Chou, R Klamroth, F Peyvandi, T Robinson, GT Group

Publikováno v: Hematology, Transfusion and Cell Therapy, Vol 46, Iss , Pp S573-S574 (2024)

Objectives: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ), a gene transfer therapy for severe hemophilia A, enables endogenous factor VIII (FVIII) production to prevent bleeding. We present efficacy and safety outcomes 4-years post-valoctocogene roxapa

Externí odkaz: https://doaj.org/article/2556db0b03f64027bd8f9cb620b1972d

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Akademický článek

Autor: FP Careta, B Madan, MC Ozelo, G Kenet, S Chou, S Pipe, A Leavitt, A Ruiz, E Dashiell-Aje, J Mahlangu

Publikováno v: Hematology, Transfusion and Cell Therapy, Vol 46, Iss , Pp S578-S579 (2024)

Objectives: Valoctocogene roxaparvovec, a gene therapy for severe Hemophilia A (HA), helps prevent bleeding by providing the body with genetic instructions for making factor VIII (FVIII) protein. We report findings from the GENEr8-1 study 4-years aft

Externí odkaz: https://doaj.org/article/79b22fc2d5ca4e6db8179cacccc517fd

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Akademický článek

ESTUDOS EM ANDAMENTO FASE IB E IIA: CROSSWALK-A E CROSSWALK-C, RANDOMIZADOS E CONTROLADOS POR PLACEBO - CROVALIMABE NO TRATAMENTO E PREVENÇÃO DE EPISÓDIOS VASO-OCLUSIVOS NA DOENÇA FALCIFORME

Autor: MA Salvino, B Antmen, FC Asirwa, S Chonat, L Franceschi, P Eleftheriou, A Inati, J Mahlangu, E Nur, S Payá-Pernía, A Charania, D Gradinaru, A Hsia, T Perretti, S Sreckovic, P Bartolucci

Publikováno v: Hematology, Transfusion and Cell Therapy, Vol 45, Iss , Pp S75-S76 (2023)

Introdução: Na doença falciforme (DF), uma mutação pontual no gene da beta-globina resulta na produção de hemoglobina S (HbS), que pode polimerizar-se após desoxigenação. A polimerização distorce a membrana dos glóbulos vermelhos e gera

Externí odkaz: https://doaj.org/article/5c67b131490642d8ab351f4534b13e7a

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Akademický článek

BLEEDING, FVIII ACTIVITY, AND SAFETY 3 YEARS AFTER GENE TRANSFER WITH VALOCTOCOGENE ROXAPARVOVEC: RESULTS FROM GENER8-1

Autor: MC Ozelo, J Mahlangu, B Madan, J Mason, F Peyvandi, AV Drygalski, G Yamaguti-Hayakawa, TM Robinson, SW Pipe, TT Gener-Eigh

Publikováno v: Hematology, Transfusion and Cell Therapy, Vol 45, Iss , Pp S451-S452 (2023)

Aims: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) provides endogenous factor VIII (FVIII) production to prevent bleeding in people with severe hemophilia A. This study evaluated bleeding, FVIII activity and safety outcomes 3 years after receiving val

Externí odkaz: https://doaj.org/article/073f51b8f2be4bc78410d269a66d55b0

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Akademický článek

Inherited bleeding disorders

Autor: N Alli, J Vaughan, S Louw, E Schapkaitz, J Mahlangu

Publikováno v: South African Medical Journal, Vol 108, Iss 1, Pp 9-15 (2018)

Abnormal bleeding is a common clinical presentation in general practice, and a rational approach to this problem is therefore required.Â Investigation of a suspected bleeding disorder necessitates a comprehensive history, thorough physical examinati

Externí odkaz: https://doaj.org/article/6f070cdf5939498c840a47c8a3cc86f6

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