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. 2016 Apr;34(4):380-3.
doi: 10.1038/nbt.3530.

Accessing the accelerated approval pathway for rare disease therapeutics

Emil D Kakkis  1 , Sara Kowalcyk  1 , Max G Bronstein  1
Affiliations

Accessing the accelerated approval pathway for rare disease therapeutics

Emil D Kakkis et al. Nat Biotechnol. 2016 Apr.
No abstract available

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References

    1. Mol Genet Metab. 2007 Sep-Oct;92(1-2):63-70 - PubMed
    1. Orphanet J Rare Dis. 2011 Jul 06;6:49 - PubMed
    1. Orphanet J Rare Dis. 2015 Feb 10;10:16 - PubMed

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