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The FDA building -- First Opinion coverage from STAT — Adobe

U.S. regulators on Wednesday approved the first treatment for an ultra-rare disease that causes people to grow bone where it otherwise shouldn’t be, extending an option to patients who have been advocating for access to the medicine.

The treatment, an oral medication that will be marketed as Sohonos, faced some questions about the quality of its data, and was rejected earlier this year by European regulators. But in approving the drug for the treatment of fibrodysplasia ossificans progressiva, or FOP, the Food and Drug Administration concluded that Sohonos’ benefits outweigh its risks.

“I just closed my eyes and looked up to the skies, like ‘Thank you, God,’” said Suzanne Hollywood, whose teenage son, Joey, has been receiving the drug in a clinical trial. “It’s far from perfect, but I’m thrilled for my son, for his future, and for the kids getting diagnosed who might not have to experience what others had to.”

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