Collaboration for rare disease drug discovery research

  • Nadia K. Litterman, Michele Rhee, David C. Swinney, Sean Ekins
  • F1000Research, October 2014, Faculty of 1000, Ltd.
  • DOI: 10.12688/f1000research.5564.1

Collaboration for rare diseases

What is it about?

This article quite simply calls for more collaboration between all parties in the rare disease ecosystem. Multiple examples are provided including Spinal Muscular Atrophy, Charcot-Marie-Tooth, Giant Axonal Neuropathy and Sanfilippo syndrome. Software for collaborations is also described.Ultimately the authors make a call for an institute for rare diseases.

Why is it important?

The authors have diverse backgrounds as either scientists or rare disease advocates and so try to put together the pieces of the puzzle that are needed for successful drug discovery in rare diseases.


Dr Sean Ekins
Collaborations in Chemistry

This was a very collaborative effort in bringing this review together, with each author contributing to make this greater than the sum of the parts. I think whats useful is we show examples of success and ongoing work that might not have been captured otherwise.

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The following have contributed to this page: Dr Sean Ekins and Dr Nadia Litterman