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Genetic disorders are diseases caused by abnormalities in the genes or chromosomes. One large class of genetic disorders is metabolic disorders, also called the ‘inborn errors of metabolism’, when a single defective gene effects the production and functioning of enzymes. Find doctors and patients dealing with genetic and metabolic disorders in our specialized support group.
The month of May saw major announcements in breakthrough therapies by companies working on the Beta-Thalassemia curatives - LentiGlobin BB305 and Luspatercept. Beta-Thalassemia is a life-threatening, blood disorder where the body fails to produce sufficient haemoglobin to thrive....Read More
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