Skinner M, Chen E, Ibrahim Q, Kucher A, Germini F, Karimi M, Clearfield E , O’Mahony B , Jain M. Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial. (Abstract). NHF (2023). Objective The Patient Reported… Continue reading Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial
Tag: Gene Therapy
Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Skinner M, Chen E, Kucher A, Germini F, Karimi M, Clearfield E , O’Mahony B , Jain M. Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE. (Abstract). NHF (2023). Introduction People with severe haemophilia A (PwSHA) commonly present with intramuscular bleeding and hemarthrosis, leading to acute and chronic… Continue reading Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Skinner M, Chen E, Kucher A, Germini F, Karimi M, Clearfield E , O’Mahony B , Jain M. Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE. (2023), CCS_Book of abstracts. Haemophilia, 29: 3-53. https://doi.org/10.1111/hae.14781 Introduction People with severe haemophilia A (PwSHA) commonly present with intramuscular bleeding and hemarthrosis,… Continue reading Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry
Naccache M, Konkle B, Peyvandi F, Miesbach W, O’Mahony B, Pile S, Youttananukorn T, Coffin D, Pierce G. Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry. (2024), POSTER ABSTRACT (MP-021). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016 Introduction The World Federation… Continue reading Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry
Long-term retention plan through myGTR – a patient engagement tool from World Federation of Hemophilia Gene Therapy Registry
Youttananukorn T, Konkle B, Peyvandi F, Naccache M, Miesbach W, O’Mahony B, Makris M, Pipe S, Skinner M, Coffin D, Pierce G.Long-term retention plan through myGTR – a patient engagement tool from World Federation of Hemophilia Gene Therapy Registry. (2024), POSTER ABSTRACT (PP-054). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016 Introduction The World Federation of… Continue reading Long-term retention plan through myGTR – a patient engagement tool from World Federation of Hemophilia Gene Therapy Registry
Gene therapy with the Padua variant of a codon-optimized human factor IX gene etranacogene dezaparvovec in people with hemophilia B: effects on patient-oriented outcomes measured using thePatient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire in the HOPE-B study
Pipe S, Abdelkader W, Clearfield E, Kucher A, Joseph B, Braverman J, Galante N, Monahan P, Ibrahim Q, Iorio A, Germini F, Skinner M. Gene therapy with the Padua variant of a codon-optimized human factor IX gene etranacogene dezaparvovec in people with hemophilia B: effects on patient-oriented outcomes measured using thePatient Reported Outcomes, Burdens and… Continue reading Gene therapy with the Padua variant of a codon-optimized human factor IX gene etranacogene dezaparvovec in people with hemophilia B: effects on patient-oriented outcomes measured using thePatient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire in the HOPE-B study
