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Opioid Use Disorder in Sickle Cell Patients with Vaso-Occlusive Crisis Linked to Greater Cost

FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in follow-up study

If the therapy is approved, Vertex has proposed a 15-year follow up of patients to evaluate the safety outcomes of the therapy. A vaso-occlusive crisis occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen and causes pain. The panel members said the 15-year follow up will help generate data from real-time monitoring of the therapy, which uses the new gene editing CRISPR technology.

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