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New Agents and Therapeutic Strategies in Beta-Thalassemia

Sponsor: educational grants from Genmab US Inc; Karyopharm Therapeutics; and Pharmacyclics LLC
Program: Satellite Symposia
Hematology Disease Topics & Pathways:
Anemias, Biological, Adult, Diseases, bioengineering, thalassemia, Therapies, cell regulation, Elderly, Genetic Disorders, red blood cells, Pediatric, Hemoglobinopathies, Biological Processes, Technology and Procedures, Young Adult, cytogenetics, Cell Lineage, erythropoiesis, gene editing, Study Population, transfusion
Friday, December 4, 2020: 3:00 PM-6:00 PM
Chair:
Janet L. Kwiatkowski, MD, MSCE, University of Pennsylvania Perelman School of Medicine
Disclosures:
Kwiatkowski: Terumo Corp: Research Funding; Apopharma: Research Funding; Bristol Myers Squibb: Consultancy; Imara: Consultancy; Celgene: Consultancy; Agios: Consultancy; bluebird bio, Inc.: Consultancy, Research Funding; Sangamo: Research Funding; Novartis: Research Funding.
Speakers:
Jeanne Boudreaux, MD, Children's Healthcare of Atlanta and Sujit Sheth, MD, Weill Cornell Medicine
Disclosures:
Boudreaux: Chiesi: Research Funding; Bluebird Bio: Consultancy. Sheth: Vertex Pharmaceuticals/CRISPR Therapeutics: Membership on an entity's Board of Directors or advisory committees; DisperSol Technologies: Research Funding; Novartis: Consultancy, Research Funding; Bluebird Bio: Consultancy; Acceleron: Consultancy; La Jolla: Research Funding; Celgene/BMS: Consultancy, Research Funding; Agios: Consultancy, Research Funding; Terumo: Research Funding.
This webinar will reveal how experts approach current therapeutic decisions for their patients with ß-thalassemia and provide a look ahead at new data and promising agents that may change practice in the near future. Renowned ß-thalassemia experts will discuss the available evidence for both transfusion-dependent thalassemia (TDT) and non-TDT, including patient case presentations, their treatment recommendations for each case, and reflection on challenges that affect therapeutic decisions. Recent clinical trial evidence and ongoing clinical trials on novel therapeutic approaches, including erythroid maturation agents and gene therapy with lentiviral vectors, will be presented and discussed, with a focus how these agents may affect management strategies for patients with ß-thalassemia in the near future. Throughout the webinar, attendees will interact with the experts through interactive case-based challenges and polling questions. Finally, attendees will be able to submit questions directly to the panel for their consideration. Discussion topics include:

  • The Medical Need for Novel Therapies for ß-Thalassemia
  • Optimizing Current Management of ß-Thalassemia
  • Evolving Role of Biologic Therapies for ß-Thalassemia
  • The Promise of Gene Therapy in ß-Thalassemia
See more of: Satellite Symposia