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A relentless pursuit.

BioMarin’s therapies are the result of a tireless drive to solve tremendous challenges.

Some individuals living with rare disease have traveled a difficult road toward diagnosis, and we are determined to provide them with options for treatment. This is evidenced by our work in phenylketonuria (PKU), a condition for which we introduced two different treatment methods where none existed before: a small molecule cofactor as well as a larger, PEGylated enzyme. Now, we are researching gene therapy in PKU to evaluate risks and potential impact. This gene therapy is under investigation and has not been determined to be safe, effective or approved for use.

Our Commitment to PKU

View BioMarin’s complete treatment portfolio here.

Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)

Learn more about PKU and BioMarin’s trailblazing legacy of developing treatment options for people living with this condition

Image: Phenylalanine Molecule

Learn more about our 15-year commitment with “The Pursuit of PKU.”

Learn More about The Pursuit of PKU