STAT Plus: BioMarin’s hemophilia gene therapy continues to prevent bleeding at four years
BioMarin’s experimental hemophilia gene therapy, which is expected to be approved by U.S. regulators in August, has continued to prevent bleeding in patients treated four years ago.
However, levels of the key clotting protein the treatment helps produce, Factor XIII, continued to slowly decline, potentially leading to questions as to how long-lasting the benefits might be.
