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Phenylketonuria

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Cerliponase alfa

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Valoctocogene roxaparvovec

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https://investors.biomarin.com/2022-02-17-BioMarin-Provides-Updates-on-Progress-in-Gene-Therapy-Programs

Wikipedia: Phenylketonuria

http://investors.biomarin.com/2017-06-01-European-Commission-Approves-Brineura-TM-cerliponase-alfa-the-First-Treatment-for-CLN2-Disease-a-Form-of-Batten-Disease-and-Ultra-Rare-Brain-Disorder-in-Children

Wikipedia: Cerliponase alfa

https://investors.biomarin.com/2017-10-26-FDA-Grants-Breakthrough-Therapy-Designation-for-BioMarins-Valoctocogene-Roxaparvovec-formerly-BMN-270-an-Investigational-Gene-Therapy-for-Hemophilia-A

Wikipedia: Valoctocogene roxaparvovec