Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are the most common monogenic diseases worldwide, with an annual diagnosis in approximately 60,000 patients with TDT and 300,000 patients with SCD. Both diseases are caused by mutations in the hemoglobin β subunit gene (HBB).…
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Herbert Needleman, MD, a physician-scientist whose lead in consumer products was removed due to research…
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