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Editorial
. 2013 Aug;21(8):1465-6.
doi: 10.1038/mt.2013.157.

Pathway for approval of a gene therapy orphan product: treading new ground

Barry J Byrne
Editorial

Pathway for approval of a gene therapy orphan product: treading new ground

Barry J Byrne. Mol Ther. 2013 Aug.
No abstract available

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References

    1. Sasinowski FJ.2011Quantum of effectiveness evidence in FDA's approval of orphan drugs. National Organization for Rare Disorders; < http://www.rarediseases.org/ docs/policy/NORDstudyofFDAapprovaloforphand... >.
    1. O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ.et al. (2013Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012 Hum Gene Ther 24355–362. - PMC - PubMed

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