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. Patients...
BioMarin Pharmaceutical’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 ...