Gene therapy for hemoglobinopathies: Current clinical applications and future perspectives
Webinar ECM FAD sincrona su terapia genica per emoglobinopatie (β-talassemia e malattia falciforme): applicazioni cliniche, gene editing, selezione del paziente e sostenibilità delle terapie avanzate.
Fonte dati per questo evento: Agenas. Evento organizzato da provider terzo. Smart ECM riporta dati pubblici dell'evento a scopo informativo e non è responsabile né del corso né dei contenuti, né ha accordi commerciali o di collaborazione con il provider.
Razionale scientifico
Gene therapy is rapidly reshaping the management of inherited hemoglobin disorders, particularly β-thalassemia and sickle cell disease (SCD). The recent clinical implementation of gene-based therapeutic strategies represents one of the most important advances in hematology and rare diseases, opening new perspectives for potentially curative treatments. Several gene therapy approaches have now entered clinical practice, demonstrating significant efficacy in reducing transfusion burden, improving quality of life, and modifying disease progression. At the same time, important challenges remain regarding patient selection, access to therapies, long-term safety, sustainability, and integration into healthcare systems. This ECM webinar aims to provide clinicians, hematologists, pediatricians, transfusion specialists, researchers, and healthcare professionals with an updated overview of the current status of gene therapy for thalassemia and sickle cell disease, with a particular focus on clinical application, patient management, and future therapeutic developments. The programme will bring together leading Italian experts actively involved in translational research and clinical implementation of advanced therapies for hemoglobinopathies. Particular attention will also be dedicated to emerging next-generation technologies, including gene editing and innovative in vivo therapeutic platforms, which are expected to further expand treatment opportunities in the coming years. The webinar intends to promote multidisciplinary discussion and strengthen scientific collaboration in the field of advanced therapies for rare hematological diseases.
Responsabili del corso
IN&FO&MED S.R.L.
Provider
ID Provider: N° 275
Città: Milano (MI)
Tipologia: Società, Agenzie Ed Enti Privati
Stato accreditamento: Standard - 1° Rinnovo
Aurelio Maggio
Responsabile scientifico
Specializzazione: Professore
Relatori e docenti
Mattia Algeri
Relatore
Juan Antonio Bueren
Relatore
Marisanta Napolitano
Relatore
Domande frequenti
L'evento è accreditato per: Medico Chirurgo (Pediatria); Biologo (Biologo); Medico Chirurgo (Medicina Interna, Ematologia); Infermiere (Infermiere); Tecnico Sanitario Laboratorio Biomedico (Tecnico Sanitario Laboratorio Biomedico).
Fonte dati per questo evento: Agenas. Evento organizzato da provider terzo. Smart ECM riporta dati pubblici dell'evento a scopo informativo e non è responsabile né del corso né dei contenuti, né ha accordi commerciali o di collaborazione con il provider.