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Sickle cell-beta thalassemia is an inherited blood disorder. The disease may range in severity from being relatively benign and like sickle cell trait to being similar to sickle cell disease . [ 1 ] [ 2 ]
[10] [11] [12] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [13] [14] [15] Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the ...
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [1] [5] [2] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.
There are two clinically significant combinations involving the sickle cell gene: Hemoglobin S/ beta thalassemia: (see above). [35] Hemoglobin S/ hemoglobin C (Hemoglobin SC disease) occurs when an individual inherits one gene for hemoglobin S (sickle cell) and one gene for hemoglobin C, The symptoms are very similar to sickle cell disease. [37]
The DSM-5 criteria puts more emphasis on social or occupational dysfunction than the ICD-10. [7] The ICD-10, on the other hand, puts more emphasis on first-rank symptoms. [2] [8] The current proposal for the ICD-11 criteria for schizophrenia recommends adding self-disorder as a symptom. [9]
Profile of a 10 year old child affected by β thalassemia, illustrating facial abnormalities. Symptoms depend on the type and severity of thalassemia. Carriers of thalassemia genes may have no symptoms (thalassemia minor), very mild symptoms with occasional crisis (thalassemia intermedia) or severe and life threatening symptoms (thalassemia major).
Generally, diseases outlined within the ICD-11 codes 6A20-6A2Z within Mental, behavioural or neurodevelopmental disorders should be included in this category. There are no pages or files in this category.
ICD-10 is the 10th revision of the International Classification of Diseases (ICD), a medical classification list by the World Health Organization (WHO). It contains codes for diseases, signs and symptoms, abnormal findings, complaints, social circumstances, and external causes of injury or diseases. [1]