Jul
14
3:30 PM15:30

14-1530E Fundraising 101

Scott R. Saylor

Are you thinking about being ACTIVE in the fight against galactosemia? Have you considered fundraising, but aren’t sure where to begin? This is the workshop for you! We want to help you be a successful DOER for the galactosemia community. This workshop is hosted by several families who have effectively planned and carried out fundraisers in the past. We will address how to pick a fundraiser, how to get started, what documents will be helpful, how to get participants, and how to attract sponsors. We hope to answer all your questions and calm your fears about organizing a fundraiser.

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Jul
14
3:30 PM15:30

14-1530B A Novel Investigational Treatment in Preclinical Development for Galactosemia

Shoshana Shendelman, PhD

Marina Brodsky, PhD

Clinical complications in galactosemia have been associated with accumulation of galactose metabolites. Preventing or reducing the formation of potentially toxic metabolites may reduce the risk of complications in galactosemia patients.   Applied Therapeutics is developing a novel investigational compound to block formation of certain galactose metabolites, which may be responsible for galactosemia complications.  Please join us to learn more about our science, treatment strategy, preclinical results and future clinical development plans.

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