What is it about?
This perspective was intended to inspire anyone to pursue testing their ideas for potential new therapeutics. Frequently very few people have access to labs, funds and facilities to test their ideas themselves so this article highlights how they might do it.
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Why is it important?
Increasingly parents of children with rare diseases and patients are taking matters into their own hands and trying to pursue treatments for their children or themselves. This article sets out how to go from idea to proof of concept and how to fund the research needed. I am not aware of others laying out a simplistic pathway that others could readily follow.
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This page is a summary of: Enabling Anyone to Translate Clinically Relevant Ideas to Therapies, Pharmaceutical Research, September 2016, Springer Science + Business Media,
DOI: 10.1007/s11095-016-2039-5.
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