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Diagnosis is made by first ruling out other causes of hemolytic anemia, such as G6PD, thalassemia, sickle-cell disease. Clinical history is also important to elucidate any underlying illness or medications that may have led to the disease. [citation needed] Laboratory investigations are carried out to determine the etiology of the disease.
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
Roland Boyd Scott (April 18, 1909 – December 10, 2002) was an American researcher, pediatrician and authority on sickle cell disease. [1] Scott authored a key paper in 1948 describing the incidence of sickle cell in infants that eventually led to the establishment of routine screening for newborns. [1]
Fadulu's research used an extract of the African "chewing stick," which he discovered, reverses sickling of red blood cells. The extract also provides protection from the disease. Medication for treating sickle cell anemia has been through the first phases of approval through the Federal Food and Drug Administration.
Tay–Sachs disease. In addition to its classic infantile form, Tay Sachs disease may present in juvenile or adult onset forms, often as the result of compound heterozygosity between two alleles, one that causes the classic infantile disease in homozygotes and another that allows some residual HEXA enzyme activity. [6] Sickle cell syndromes. A ...
Stephanie was born with sickle-cell disease. At the age of 5, she participated in a clinical trial at St. Jude Children's Research Hospital, which used bone marrow transplants to treat the disease. The treatment was highly effective for her, and Dr. Keith Wagner, who led the study, considered Stephanie one of its most successful cases.
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