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.