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 carry stratospheric prices – a prospect already worrying consumer advocates and economists.
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