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BioMarin Pharmaceutical’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Topic Hubs AI drug discovery: News, analysis and interviews Antibody-drug conjugates: The next wave of targeted cancer drugs Biopharma M&A and dealmaking: Who’s buying and why Biopharma people moves: CEO changes, executive hires, board positions Biotech IPOs: Tracking the market for new public offerings Biotech venture funding: Who’s raising and where the money is going Cell and gene therapy manufacturing: Scaling the next frontier China biotech: Licensing deals and the Biosecure Act CRISPR and gene editing: The push for in…
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 ...
Source: Endpoints News