Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring doubts about its approval prospects.
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Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring doubts about its approval prospects.
Published May 19, 2026
A BioMarin Pharmaceutical sign hangs on the side of an office building in Brisbane, California, on Sept. 21, 2020.
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