BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test
BioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don't produce enough of the ENPP1 enzyme, which generates plasma inorganic pyrophosphate ...
May 19, 2026 06:18 AM EDTUpdated 06:35 AM R&D BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test Reynald Castaneda Deputy Editor BioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don’t produce … Sign up to read this article for free. Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.
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