11. Sickle cell disease describes a group of inherited disorders, including sickle cell anemia and sickle B-thalassemia. 12. Sickle cell disease is an inherited disorder in which oxygen-carrying red blood cells are defective. 13. Some of the new therapeutics are narrowly focused on helping certain patients who failed other medications or have rare inherited disorders. 14. The Amish, like other close ethnic groups around the world, suffer high rates of inherited disorders because of the sudden emergence of recessive genes passed through generations. 15. The decision to begin an initial safety study of the drug, named Lumarel, follows eight attempts to treat the inherited breathing disorder using gene therapy. 16. The patients, two young children, suffered from a rare inherited disorder that left them without a functioning immune system. 17. The remaining clinical trial at the medical center involved patients with hemophilia, the inherited bleeding disorder. 18. There is growing evidence that even people without an inherited disorder can develop problems associated with iron overload. 19. PKU is an inherited disorder that, if left untreated, can lead to mental retardation, seizures and other symptoms. 20. An inherited bleeding disorder appears to be a common cause of heavy menstrual periods and treating it could reduce the need for hysterectomies, according to research published Friday. |
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