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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
The new gene therapies for sickle cell disease—including the gene-editing treatment Casgevy ... a pill that could do what the genetics do, that would be great." In new work just published ...
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Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
The reimbursement agreement comes as the National Institute for Health and Care Excellence (NICE) issues positive guidance recommending CASGEVY ... industry's top places to work, including ...