Pointing to a cure, BioMarin reports impressive trial results for hemophilia A gene therapy
BioMarin says it nailed down hard evidence that its gene therapy for hemophilia A demonstrated a high chance of restoring patients’ natural clotting abilities, reducing or eliminating bleeding episodes and pointing them down the road to a normal life. And company $BMRN investigators say they are now hustling BMN 270 into a Phase IIb study that could be used to gain an accelerated approval for a potential blockbuster.
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