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In their groundbreaking Nature Communications paper, scientific pioneers Lon Cardon and Karol Estrada shed new light on the decades-old question of how to identify optimal drug targets

Read through the history, achievements, and milestones of BioMarin.

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Know the achievements of our Hemophilia Community of patients and caregivers through arts and self-expression.

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Read the story of MPS IVA patient, Annabelle affected by rare genetic disease. Know the struggle, fight & willingness to tackle.