Small companies focused on rare and neglected diseases
What is it about?
A short article on how a scientist met the parent of a child with a rare disease and it lead to them forming a company, connecting with scientists, writing and obtaining grants and developing a treatment. This in turn lead to the realization that this could be replicated elsewhere for other rare diseases as well as neglected diseases.
Why is it important?
The perspective is important because it focuses on the incentive of the pediatric rare disease priority review voucher and to some extent the neglected tropical disease voucher. For rare diseases where there are very few patients - the only likely return on investment is the chance of getting a voucher.
The following have contributed to this page: Dr Sean Ekins