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Advocating for SDS Research at Rare Drug Development Symposium

From the Founder, Dr. Eszter Hars, Ph.D.


This week, I had the great honor to be invited as a speaker and panelist to present at the 2021 Rare Drug Development Symposium, hosted by Global Genes and the University of Pennsylvania Orphan Drug Center.

In my talk, I raised awareness about Shwachman-Diamond Syndrome and its effect on our patients, highlighted the critical importance of collaboration and patient engagement, and the need for the patient community to coordinate research and therapy development efforts with professionalism, focus, end efficiency using our new mouse model project as an example.

The session was for young researchers to discuss how to make the best out of working with rare diseases communities and ways to collaborate, and concluded with a panel discussion with my co-presenters Bina Shah, MBA; Amy Waldman, M.D., M.S.C.E.; and session moderator Benjamin Forred, Ph.D.

All sessions were recorded and are now available on the conference platform to all registered users. The recording will also be made available to the public at the end of summer/early fall on the Global Genes resources. I will continue to advocate for SDS and make SDS an attractive area of research for researchers and biotech companies.




To support the SDS Alliance's research and therapy development initiatives - starting with the mouse model project - please donate today and help us fundraise. (For international supporters, we have set up a Facebook fundraiser that doesn't charge credit card fees.)
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