Release Date: January 14, 2025
Expiration Date: January 14, 2026
Activity Overview
The most common monogenic global disorders worldwide are hemoglobinopathies, which are genetic diseases related to hemoglobin synthesis. The genetic cause of hemoglobinopathies is DNA variants in or near the globin genes. Approximately 7% of the global population carries a DNA variant, with the majority of diagnosed individuals having sickle cell disease (SCD); the minority of patients are affected by β-thalassemia. Although hemoglobinopathies are diverse, their burden is severe and disabling for many individuals and requires specialized multidisciplinary management. This continuing medical education symposium features engaging, case-based discussions among 3 experts, highlighting clinical trial data for gene therapies and strategies for integrating gene therapy into the individualized management of hemoglobinopathies including SCD and transfusion-dependent thalassemia.
This educational activity is an archive of the live/virtual symposium held on December 6, 2024.
Target Audience
This educational activity is directed toward pediatric and adult hematologists, hematologist-oncologists, pediatricians, nurses, nurse practitioners, physician assistants, and other clinicians involved in the management of patients living with hemoglobinopathies.
Learning Objectives
Upon successful completion of this activity, you should be better prepared to:
- Describe the burden of disease of transfusion-dependent thalassemia (TDT)
- Outline the burden of disease of sickle cell disease (SCD)
- Formulate individualized management plans for patients with TDT
- Design personalized treatment plans for patients with SCD
- Evaluate clinical trial data for gene therapy for hemoglobinopathies
- Integrate gene therapy into management plans for patients with hemoglobinopathies
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