In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease. The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for haemophilia, sickle-cell anaemia, and an array of other genetic diseases. Yet the products, should they reach patients, are likely to cost as much as $1 million for both eyes.
FDA Just Approved The First-Ever Gene Therapy For an Inherited Disease
FDA Just Approved The First-Ever Gene Therapy For an Inherited Disease