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. 2019 Apr;37(4):352-355.
doi: 10.1038/s41587-019-0081-7.

What compassionate use means for gene therapies

Carolyn Riley Chapman  1 , Kenneth I Moch  2 , Andrew McFadyen  3 , Lisa Kearns  4 , Tom Watson  5 , Pat Furlong  6 , Alison Bateman-House  4
Affiliations

What compassionate use means for gene therapies

Carolyn Riley Chapman et al. Nat Biotechnol. 2019 Apr.
No abstract available

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References

    1. U.S. Department of Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Long term follow-up after administration of human gene therapy products: draft guidance for industry. https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-bio-gen/documents/d... (2018).
    1. MIT NEWDIGS Initiative. FoCUS Project: financing of cures in the US. Research Brief 2017F211.v011. https://newdigs.mit.edu/sites/default/files/FoCUS_Research_Brief_2017F21... (2017).
    1. U.S. Food and Drug Administration. Expanded access. https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassio... (25 August 2017).
    1. Caplan, A. & Moch, K. Rescue me: the challenge of compassionate use in the social media era. Health Affairs Blog https://www.healthaffairs.org/do/10.1377/hblog20140827.041027/full/ (2014).
    1. Borysowski, J., Ehni, H. & Górski, A. BMC Med. 15, 136 (2017). - DOI

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