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There’s huge excitement over the potential of gene therapy. But one-time treatments will only be successful if they provide long-term benefit for patients.

For BioMarin Pharmaceuticals (BMRN), soon-to-be-released data could offer indications about just how durable its gene therapy against hemophilia A could be. In June, the Novato, Calif.-based biotech is expected to disclose an update from a clinical trial that has followed the treatment of patients for three years.


Here are nine things to know about BioMarin’s gene therapy and this crucial measure of its long-term efficacy.

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