Biomarin to seek approval for hemophilia gene therapy but doubts persist

Biomarin to seek approval for hemophilia gene therapy but doubts persist

STAT Plus: Biomarin to seek approval for hemophilia gene therapy, but durability questions persist

By ADAM FEUERSTEIN AND MATTHEW HERPER

ADOBE

The company said that an experimental gene therapy targeting hemophilia A continued to help patients produce a protein enabling blood to clot.

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