Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases.
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E.K. is a current employee of and stockholder in Ultragenyx, a stockholder in BioMarin and president of the EveryLife Foundation for Rare Diseases.
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Kakkis, E., Kowalcyk, S. & Bronstein, M. Accessing the accelerated approval pathway for rare disease therapeutics. Nat Biotechnol 34, 380–383 (2016). https://doi.org/10.1038/nbt.3530
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DOI: https://doi.org/10.1038/nbt.3530