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Home » BioMarin Secures Orphan Drug Status for BMN 270 Gene Therapy
BioMarin Secures Orphan Drug Status for BMN 270 Gene Therapy
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The FDA has awarded BioMarin orphan drug designation for its experimental gene therapy BMN 270 for the treatment of hemophilia A.
It is being studied in a five-year Phase 1/2 clinical trial in up to 12 patients with severe hemophilia A.
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