Virtual collaborations for developing Sanfilippo syndrome treatments on a shoestring

  • Sean Ekins, Derek R. Moen, Jill Wood
  • Molecular Genetics and Metabolism, February 2016, Elsevier
  • DOI: 10.1016/j.ymgme.2015.12.247

a small company for Sanfilippo syndrome

What is it about?

This describes our work to date building Phoenix Nest, Inc. into a small company focused on Sanfilippo syndrome.

Why is it important?

This disease is ultra rare and the forms we have focused on MPS IIIC and IIID affect very small numbers and therefore few if any companies will work on a treatment or cure.


Dr Sean Ekins
Collaborations in Chemistry

Our approach might inspire others to take the same route. NINDS recently told us at least they know of one company that followed what we did and started a small company for their rare disease. Perhaps more will follow.

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The following have contributed to this page: Dr Sean Ekins