Patients with the ultra-rare disease FOP grow bone where it doesn’t belong, creating a life-threatening second skeleton. Regeneron Pharmaceuticals believes it can offer hope: A new treatment reduced new bone growth by 90% in a clinical trial, the company said, a result that could lead to Food and Drug Administration approval.

But convincing the FDA will be a matter of explaining a complicated study of a first-of-its-kind drug, targeting a disease that affects only about 800 people and has no approved treatments.

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