Stop Sanfilippo
Stop Sanfilippo is an organization dedicated to the promotion of research against Sanfilippo Syndrome and the care of affected children.
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Abeona Therapeutics
Grant in 2017
Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy-based treatments for severe and life-threatening rare genetic diseases. Its pipeline includes therapies for Sanfilippo syndrome types A and B, epidermolysis bullosa, Batten disease, cystic fibrosis, and genetic eye disorders.
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