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FDA Approves Breakthrough Sickle Cell Treatments

By Genoa Barrow | OBSERVER Senior Staff Writer - The FDA has approved the first two gene therapies to treat sickle cell disease, providing hope for patients, their families, and the entire sickle cell community, but the treatments are expensive and require specialized care to be effective.

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FDA approves first gene therapies for sickle cell disease

FDA approves first gene therapies for sickle cell disease
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FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.

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