A breakthrough treatment for sickle cell patients could soon become the first gene-editing treatment to be approved by the Food and Drug Administration (FDA). Sickle cell disease (SCD) is an inherited blood disorder that affects roughly 100,000 Americans, most of whom are Black. The disease causes red blood cells to become sickle or c-shaped, clumping together and clogging blood vessels, causing pain and other complications. There is…
Experts emphasize the fundamental principles of integrative care, the success seen in treating rare diseases, and how it can be applied to all chronic diseases.
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/PRNewswire/ This week, the National Organization for Rare Disorders (NORD®) hosted the virtual Rare Diseases and Orphan Products Breakthrough Summit,.