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Accessing the accelerated approval pathway for rare disease therapeutics

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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Correspondence to Emil D Kakkis.

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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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