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Publications

Following the science is one of BioMarin’s core principles, and we believe sharing our discoveries with the research and medical communities is critical to help improve our collective understanding of how to use genetics to inform the treatment of serious medical conditions. Our findings have been published in many top-tier medical journals. Access some of our publications below.

This page contains a non-exhaustive selection of publications provided to support scientific exchange with scientists and researchers. Any content about investigational therapies or investigational uses of approved products does not establish the safety or efficacy of these therapies or uses. BioMarin does not recommend or suggest use of its medicines in a manner inconsistent with approved labeling. Clinical study results are intended to report the results of the study that were known at the time of the study’s completion and do not provide a comprehensive or current review of the safety and efficacy of any drug based upon all information available. Review the methodology, limitations and author’s financial disclosures for additional information. This information is not medical advice and should not replace independent judgment and additional research. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned for information about its indication and important safety information.

Featured Publication

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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BioMarin Publication

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