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.
About the Author Reprints
I see on twitter you said you wrote a preview for (3) readouts. $BCRX $BMRN $IFRX can you show me where it is on this website? THANKS!
The links were in Adam’s tweet, Harold. Here they are again:
Pat Skerrett
STAT