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The US Food and Drug Administration has approved two new breakthrough therapies for patients with sickle cell disease (SCD), and we believe both may enhance the treatment of our patients here at CHOP.
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The US Food and Drug Administration has granted accelerated approval to two novel drugs to treat sickle cell disease.
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Stefano Rivella, PhD, an expert in creating the next-generation lentiviral vectors for the cure of hemoglobinopathies including sickle cell disease (SCD), as well as a leader in the discovery of erythroid and iron disorders including beta-thalassemia, has led three new published studies that make significant contributions to the scientific body of literature regarding red blood cells, and how manipulating the production of red cells can lead to the development of new disease-modifying therapies.
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Born with sickle cell disease, Naomi receives breakthrough treatments and screenings at CHOP to keep her symptoms in check and protect her from strokes.
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Researchers have found a key protein that could become a target for a new treatment for sickle cell disease. In lab tests, blocking the protein reduces sickling that distorts red blood cells.
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Proceeds support sickle cell research and patient care at Children’s Hospital of Philadelphia.
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Read advice for working with schools to keep your child with chronic disease healthy and safe.
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CHOP scientists altered red blood cells to produce a form of hemoglobin not affected by the mutation that causes sickle cell disease. The findings may lead to a new therapy for the disease.