WSJ - US Business

FDA Blesses Blindness Treatment That Could Cost $1 Million

The Food and Drug Administration approved for sale the first therapy in the U.S. that delivers a functional gene to replace a faulty, disease-causing one—a treatment that could carry a price in excess of $ 1 million, its maker has said.

The injected gene therapy from Spark Therapeutics Inc. is designed to improve sight in people with a rare form of vision loss caused by an inherited genetic mutation. The condition, retinal dystrophy, often manifests itself in young children and affects up to 3,000 Americans, Spark said.


WSJ.com: US Business

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