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Research & Development

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Family Camp Helps Create Community, Support Networks for People with PKU

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Supplier Diversity

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BioMarin Pioneer Honored through New Building Name and Book Launch

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Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Angela Schulz, M.D., Temitayo Ajayi, M.D., Nicola Specchio, M.D., Ph.D., Emily de Los Reyes, M.D., Paul Gissen, M.B., Ch.B., Ph.D., Douglas Ballon, Ph.D., Jonathan P. Dyke, Ph.D., Heather Cahan, M.D., Peter Slasor, Sc.D., David Jacoby, M.D., Ph.D., and Alfried Kohlschütter, M.D., for the CLN2 Study Group

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BioMarin Community Guidelines

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It’s Time to Give a Platform, or in My Case “Runway,” to People with Rare Disease

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MPS IVA

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Terms of Use – EU (Deutsch)

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From Height to Health: Changing the Conversation in Achondroplasia

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Giving

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Patient Community Engagement

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Barbara Bodem

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Expanded Access

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Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Johnny Mahlangu, M.B., B.Ch., M.Med., Radoslaw Kaczmarek, Ph.D., Annette von Drygalski, M.D., Pharm.D., Susan Shapiro, M.D., Ph.D., Sheng-Chieh Chou, M.D., Margareth C. Ozelo, M.D., Ph.D., Gili Kenet, M.D., Flora Peyvandi, M.D., Ph.D., Michael Wang, M.D., Bella Madan, M.D., Nigel S. Key, M.D., Michael Laffan, D.M., Ph.D., et al., for the GENEr8-1 Trial Group*

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Valoctogene Roxaparvovec Gene Therapy for Hemophilia A

Margareth C. Ozelo, M.D., Ph.D., Johnny Mahlangu, M.B., B.Ch., M.Med., K. John Pasi, M.D., Ph.D., Adam Giermasz, M.D., Ph.D., Andrew D. Leavitt, M.D., Michael Laffan, M.D., Ph.D., Emily Symington, M.D., Doris V. Quon, M.D., Ph.D., Jiaan-Der Wang, M.D., Ph.D., Kathelijne Peerlinck, M.D., Ph.D., Steven W. Pipe, M.D., Bella Madan, M.D., et al., for the GENEr8-1 Trial Group*

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The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

P. Harmatz, C.J. Hendriksz, C. Lampe , J.J. McGill, R. Parini, E. Leão-Teles, V. Valayannopoulos , T.J. Cole , R. Matousek, S. Graham , N. Guffon, A. Quartel, MPS VI Study Group