Key Points
Vertex and Crispr Therapeutics will need to provide more data on the therapys safety and efficacy for it to remain available...
Both sickle cell disease and beta thalassemia are genetic conditions caused by errors in the genes for hemoglobin, the vital protein found in red blood cells that carries oxygen throughout the body..
Sickle cell disease disproportionately affects people of African and Caribbean descent, while beta thalassemia mainly affects people of Mediterranean, South Asian, Southeast Asian, and Middle Eastern origin...
Both sickle cell disease and beta thalassemia are painful, lifelong conditions that in some cases can be fatal, said Julian Beach, interim executive director of healthcare quality and access at the UKs MHRA, on Thursday...
Afterward, they may need to spend a month or more in a hospital while the edited cells take up residence in the bone marrow and start to make healthy red blood cells...
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