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FDA Approves Gene Therapy for Younger Children With Sickle Cell Disease

The US Food and Drug Administration (FDA) issued supplemental approval of a gene therapy for patients aged 2 years or older with either sickle cell disease and recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia.

We haven't written up this one. JAMA has the full story — the link below goes straight to it.

Read the original at jamanetwork.com →

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