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study of biallelic variants in cyp2r1 causing vitamin d hydroxylation deficiency rickets type 1b
Original title: “Study of biallelic variants in CYP2R1 causing vitamin D hydroxylation deficiency rickets type 1B.”
BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
Original title: “BioMarin suffers another blow to rare disease portfolio in Phase 3 flop”