Now, more than 5000 American children with sickle cell disease can access a genetic cure for a disease that causes pain, organ damage, loss of school days, ans limited participation in sports and other childhood activities.

Possibly more important: The FDA’s action on Casgevy/Sickle-Cell Disease suggests a more liberal view of extending to pediatric patients other genetic cures until now reserved for adults. Since the vast majority of genetic diseases first appear in infants, this FDA action may encourage more investment in public and private biopharmas working on agents for other genetic diseases first seen in childhood.