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.
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.