Sickle Cell Gene Therapy Approved for Children as Young as Two

“The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals’ gene therapy in children as ​young as two with inherited blood disorders, including ‌sickle cell disease, the first such treatment cleared for this age group. Casgevy, a one-time treatment made from a patient’s own blood stem ​cells, was previously approved for patients aged […] The post Sickle Cell Gene Therapy Approved for Young Children appeared first on Human Progress.