denotes an abstract that is clinically relevant.
denotes that this is a recommended PHD Trainee Session.
denotes that this is a ticketed session.Sponsor: Vertex Pharmaceuticals
Program: Friday Satellite Symposia
Hematology Disease Topics & Pathways:
Sickle Cell Disease, Thalassemia, Education, Hemoglobinopathies, Diseases
Hematology Disease Topics & Pathways:
Sickle Cell Disease, Thalassemia, Education, Hemoglobinopathies, Diseases
Friday, December 6, 2024: 11:00 AM-2:00 PM
Room 30
(San Diego Convention Center)
Chair:
Biree Andemariam, MD, University of Connecticut Health
Disclosures:
Andemariam: American Society of Hematology: Research Funding; Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; Editas: Other: Data Safety Monitoring Board / Adjudication Committee; Sanofi Genzyme: Consultancy, Membership on an entity's Board of Directors or advisory committees; Hemanext: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Fulcrum Therapeutics: Other: Data Safety Monitoring Board / Adjudication Committee; Health Resources and Services Administration: Research Funding; Vertex: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Agios: Consultancy, Membership on an entity's Board of Directors or advisory committees; Connecticut Department of Public Health: Research Funding; Roche: Consultancy, Membership on an entity's Board of Directors or advisory committees; Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; bluebird bio: Consultancy, Membership on an entity's Board of Directors or advisory committees; Afimmune: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding; Accordant: Consultancy, Membership on an entity's Board of Directors or advisory committees.
Speakers:
Farzana Sayani, MD, MSc, University of Pennsylvania
and
Sujit Sheth, MD, Weill Cornell Medicine
Disclosures:
No relevant conflicts of interest to declare.
The most common monogenic global disorders worldwide are hemoglobinopathies, genetic diseases related to hemoglobin (Hb) synthesis. The genetic cause of hemoglobinopathies are DNA variants in or near the globin genes. It has been estimated that 7% of the global population carry a DNA variant, with the majority of diagnosed individuals having sickle cell disease, and the minority are affected by β-thalassemia. Though diverse, the burden of hemoglobinopathies is known to be severe and disabling for many individuals, requiring specialized multidisciplinary management. This accredited symposium will feature engaging, case-based discussions among 4 experts highlighting clinical trial data for gene therapies and strategies for integrating gene therapy into the individualized management of hemoglobinopathies, including sickle cell disease and transfusion-dependent thalassemia.
See more of: Friday Satellite Symposia