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Report an Adverse Event

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Reporting a Product Complaint

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Global Compliance and Ethics Program

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Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study

Gili Kenet, Yeu-Chin Chen, Gillian Lowe, Charles Percy, Huyen Tran, Annette von Drygalski, Marc Trossaert, Mark Reding, Johannes Oldenburg, Maria Eva Mingot-Castellano, Young-Shil Park, Flora Peyvandi, Margareth C. Ozelo, Johnny Mahlangu, Jennifer Quinn, Mei Huang, Divya B. Reddy, Benjamin Kim

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BioMarin Pioneer Honored through New Building Name and Book Launch

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Clinical Trial Diversity

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Learning and Development

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Revealing the clinical phenotype of atypical neuronal ceroid lipofuscinosis type 2 disease: Insights from the largest cohort in the world

Charles M Lourenço, Andre Pessoa, Carmen C Mendes, Carolina Rivera-Nieto, Diane Vergara, Mónica Troncoso, Emily Gardner, Francisca Mallorens, Lina Tavera, Luis A Lizcano, Nora Atanacio, Norberto Guelbert, Norma Specola, Nury Mancilla, Carolina FM de Souza and Sara E Mole

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Jean-Jacques Bienaime

• Leadership Profile

Henry Fuchs, MD

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Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data

Melissa Lah, Keziah Cook, Dumingu Aparna Gomes, Stephanie Liu, Nadia Tabatabaeepour, Noam Kirson, Er Chen, Kristin Lindstrom, Kaleigh Bulloch Whitehall, Joost Van Backle, Barbara K. Burton

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PKU

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External Scientific Support

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Giving

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Patient Community Engagement

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Lifetime Impact Study for Achondroplasia (LISA): Findings from an observational and multinational study focused on health-related quality of life in individuals with achondroplasia in Latin America

Juan Llerena Jr, Pablo Rosselli, Amanda Aragao, Cristina Valenzuela, Debora Bertola, Yaneth Mendez, Mariana del Pino, Nicolette Calvacanti, Paula Thomazinho, Jeanne M. Pimenta, Shelda Cohen, Tom Butt, Jose C. Thomaz Jr, Renee Shediac , Richard Rowell, Tatiana S.P.C. Magalhaes, Chong Kim, Virginia Fano

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Supporting Women at BioMarin: An Interview with the Co-Chairs of Our Women’s Leadership Development Group

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MPS IVA

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International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria

Ania C. Muntau, Darius J. Adams, Amaya Bélanger-Quintana, Tatiana V. Bushueva, Roberto Cerone, Yin-Hsiu Chien, Ana Chiesa, Turgay Coşkun, Javier de las Heras, François Feillet, Rachel Katz, Florian Lagler, Flavia Piazzon, Fran Rohr, Francjan J. van Spronsen, Paula Vargas, Gisela Wilcox, Kaustuv Bhattacharya

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Management of early treated adolescents and young adults with phenylketonuria: Development of international consensus recommendations using a modified Delphi approach

Barbara K. Burton, Álvaro Hermida, Amaya Bélanger-Quintana, Heather Bell, Kendra J. Bjoraker, Shawn E. Christ, Mitzie L. Grant, Cary O. Harding, Stephan C.J. Huijbregts, Nicola Longo, Markey C. McNutt II, Mina D. Nguyen-Driver, André L. Santos Pessoa, Júlio César Rocha, Stephanie Sacharow, Amarilis Sanchez-Valle, H. Serap Sivri, Jerry Vockley, Mark Walterfang, Sarah Whittle, Ania C. Muntau