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A hemophilia patient hero at zero, BioMarin to seek FDA OK for gene therapy. But will the effect last?
San Francisco Business Times

A potential one-shot-and-you’re-done hemophilia A treatment from BioMarin Pharmaceutical Inc. will target regulatory approval in the United States and Europe after showing dramatic decreases in bleeding during clinical trials.

But in a competitive market to find the next big and expensive treatment to help — or even cure — hemophilia A patients, questions linger around how long the treatment sticks with patients. BioMarin (NASDAQ: BMRN) apparently didn’t allay those concerns Tuesday: The San Rafael-based company said its treatment appeared to plateau in restoring the amount of a clotting protein, known as Factor VIII.