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.
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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.
Perspectives
This is about as close as you get to writing about what you have been doing in the background for several years. In the case of Phoenix Nest it highlights the persistence paying off and getting an STTR after many submissions. A wonderful opportunity to highlight Sanfilippo syndrome and gain more visibility for the disease.
Dr Sean Ekins
Collaborations in Chemistry
Read the Original
This page is a summary of: Incentives for Starting Small Companies Focused on Rare and Neglected Diseases, Pharmaceutical Research, December 2015, Springer Science + Business Media,
DOI: 10.1007/s11095-015-1841-9.
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