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BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test
BioMarin Pharmaceutical’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study.
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biopharmadive.com
BioMarin drug acquired in buyout misses goal in rare disease study
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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endpoints.news
BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test
BioMarin Pharmaceutical’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study.