FDA Approves Gene Therapy for Sickle Cell – What You Need to Know

In a groundbreaking development, federal officials have approved two gene therapy treatments designed to address sickle cell disease. This approval represents a major milestone in the realm of genetic medicine and is particularly significant for the tens of thousands of individuals afflicted by this inherited blood disorder. Sickle cell disease, marked by excruciating pain crises and early mortality, has long been in the shadows of medical research. The recent approval by the Food and Drug Administration (FDA) signals a transformative moment for those grappling with the severe impacts of this condition. While it is premature to declare patients “cured,” 95 percent of recipients are now symptom-free, marking a remarkable success in the realm of genetic medicine. Choosing Gene Therapy In the journey towards combating sickle cell disease, the decision to undergo gene therapy is a deeply personal one, shaped by the unique experiences and considerations of each patient. Both Jennelle Stephenson and Victoria Gray, among the early recipients of sickle cell gene therapy, made a conscious choice to embark on this groundbreaking medical path, driven by a combination of factors. Courtesy Jennelle Stephenson/USA Today Jennelle Stephenson’s Leap of Faith For Jennelle Stephenson, the choice to pursue gene therapy was a...