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Patient Support

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Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy

Todd Oppeneer, Yulan Qi, Joshua Henshaw, Kevin Larimore, Andrew Melton, Jukka Puoliväli, Caitlyn Carter, Pierluigi Fant, Sebastian Brennan, Laura A. Wetzel, Monika A. Sigg, Brett E. Crawford, Jenna Magat, Steven Froelich, Josh C. Woloszynek, Charles A. O’Neill

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BioMarin Fellowships

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BioMarin to Host First Quarter 2025 Financial Results Conference Call and Webcast on Thursday, May 1, 2025, at 4:30pm ET

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Achondroplasia Clinical Trials

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BioMarin to Present at the TD Cowen 45th Annual Healthcare Conference on Tuesday, March 4 at 6:50 AM PT / 9:50 AM ET in Boston, MA

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BioMarin Appoints Pioneer in Personalized Medicine, Dennis J. Slamon, M.D., Ph.D., to Board of Directors

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BioMarin Announces New England Journal of Medicine Publishes Vosoritide Phase 2 Study Showing Sustained Annualized Growth Up to 42 Months in Children with Achondroplasia

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New England Journal of Medicine Published Open-label Study Showing Brineura® (cerliponase alfa) Reduced the Rate of Clinical Decline of Children with CLN2 Disease, a Form of Batten Disease

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BioMarin Announces New England Journal of Medicine Publishes 3 Years of Follow-up Data in Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

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BioMarin Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A

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BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A

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Research & Development

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Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

Glenn F. Pierce, Sylvia Fong, Brian R. Long, Radoslaw Kaczmarek

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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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Experiences of children and adolescents living with achondroplasia and their caregivers

Renée Shediac, Olga Moshkovich, Heather Gerould, Rachel Ballinger, Agnes Williams, M. Alex Bellenger, Jennifer Quinn, Julie Hoover-Fong, Klaus Mohnike, Ravi Savarirayan, Dominique Kelly

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BioMarin Pioneer Honored through New Building Name and Book Launch