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Nat Biotechnol. 2014 Jun;32(6):515-8. doi: 10.1038/nbt.2924.

Expanding rare disease drug trials based on shared molecular etiology.

Author information

1
Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.
2
Office of Special Initiatives, Office of the Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.
PMID:
24911489
PMCID:
PMC4548299
DOI:
10.1038/nbt.2924
[Indexed for MEDLINE]
Free PMC Article

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