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BLEEDING RATE, FVIII UTILIZATION, CORTICOSTEROID USE, QUALITY OF LIFE AND SAFETY 4-YEARS AFTER GENE TRANSFER WITH VALOCTOCOGENE ROXAPARVOVEC IN A BRAZILIAN COHORT: RESULTS FROM GENER8-1
MC Ozeloa,b, GGY Hayakawaa,b, ANL Prezottic, MH Cerqueirad, PR Villaçae, C Lorenzattof, T Robinsong
a Centro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, Brazil
b Departamento de Medicina Interna, Faculdade de Ciências Médicas, Universidade de Campinas (UNICAMP), Campinas, SP, Brazil
c Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos (HEMOES), Vitória, ES, Brazil
d Hemocentro do Estado do Rio de Janeiro (HEMORIO), Rio de Janeiro, RJ, Brasil
e Hospital de Clínicas, Faculdade de Medicina da Universidade de São Paulo (USP), São Paulo, SP, Brazil
f Centro de Hematologia e Hemoterapia do Paraná (Hemepar), Curitiba, PR, Brazil
g BioMarin Pharmaceuticals Inc, Novato, CA, USA
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ISSN: 25311379
Original language: English
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Idiomas
Hematology, Transfusion and Cell Therapy