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BioMarin’s rare disease therapy shows no clinical benefit in Phase 3 test
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 … Read More