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- BioMarin's Phase 3 trial of ENPP1 enzyme therapy fails to show benefitBioMarin reported that its late-stage study of an enzyme replacement therapy for ENPP1 deficiency did not meet its primary efficacy endpoints. The trial, which enrolled patients lacking the ENPP1 e…
- Wave Life Sciences reports early-stage data showing monthly dosing possible for RNA editing therapy in AATDWave Life Sciences announced an updated cut of data from its early-stage study of an RNA editing treatment aimed at alpha‑1 antitrypsin deficiency (AATD). The new data suggest the genetic therapy…