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.
The following have contributed to this page: Dr Sean Ekins