Lenmeldy - The World’s Most Expensive Gene Therapy

Lenmeldy - The World’s Most Expensive Gene Therapy

TIME

The one-time treatment, Lenmeldy, won U.S. regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD. The drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday. It affects about one in 100,000 live births, and fewer than 40 children a year.

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