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0819 biomarin hemophilia
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Regulation

What FDA’s request for a new endpoint means for BioMarin’s hemophilia gene therapy

Collecting two-year data will set BioMarin back at least 15 months, but it still has a shot at getting its gene therapy to market first

Collecting two-year bleeding rates from all patients will set BioMarin back at least 15 months, but it still has a good shot at getting its gene therapy to market first.

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