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Drug vials Payment options for the drug include spreading out the costs over time, refunds for patients whose treatment fails and discounts for insurers that provide swift coverage. (Photo: Shutterstock)

A potential cure for a lethal childhood disorder — the first of its kind in the U.S. — is hitting the market at a cost of $2.1 million, paving the way for more therapies that bring dramatic benefits for patients, along with challenges for health-care systems.


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