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Which Treatment Is Right for Me or My Child?

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source: Children's National Hospital

year: 2020


Overview of Presentation:
• Background: Timeline of Therapies in SCD
• Background: Complications in Sickle Cell Disease
• Review of current disease modifying therapies and clinical trials
• Review of new mechanisms of action for new disease modifying therapies
• Considerations and Clinical Scenarios for therapies in SCD
• Review current FDA approved treatments and Curative therapies

Future for new Therapies Are Bright for SCD!
• There are over 20 new therapeutic products under investigation for SCD
• These include Bone Marrow Transplant and Gene Therapies


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