Gene therapy for children with sickle cell disease approved by FDA

CRISPR gene therapy insights may improve access to sickle cell cures

First New York patient cured of sickle cell disease using Lyfgenia gene therapy

Sebastien Beauzile, a 21-year-old living with sickle cell anemia, has become the first person in New York State to be cured of the disease u

WHO publishes first global guidelines for managing sickle-cell disease in children and adolescents

This article presents the first WHO normative guideline on the diagnosis, prevention, and clinical management of sickle-cell disease (SCD) i

First Louisiana patient functionally cured of sickle cell disease through gene therapy

A man in Louisiana has become the first person in the state to be functionally cured of sickle cell disease through gene therapy. He celebra

FDA reverses course on Regenxbio’s childhood gene therapy after rejection

High-cost gene therapies face financing barriers in US healthcare system

Gene therapies costing $2 million or more per patient can cure diseases like sickle cell with a single treatment, but America's healthcare s