![]() Helen O’Neill of University College London. “The future of life-changing cures resides in CRISPR based (gene-editing) technology,” said Dr. To date, bone marrow transplants, extremely arduous procedures that come with very unpleasant side effects, have been the only long-lasting treatment. Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. In a statement Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020. ![]() LONDON - Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K.
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