The devastating diagnosis of a rare disease is prompting an increasing number of people to go to the bench and take research into their own hands
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21 November 2019
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
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Branca, M.A. Their lives in their hands. Nat Biotechnol 37, 1255–1260 (2019). https://doi.org/10.1038/s41587-019-0303-z
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DOI: https://doi.org/10.1038/s41587-019-0303-z
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A patient advocating for transparent science in rare disease research
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