BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test

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. SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER