Sanfilippo Syndrome (MPSIIIA) PerlQuest
“The opportunity to speed the time from drug discovery to patient access through Perlara’s screening platform is something we are thrilled to support. Neurodegeneration in Sanfilippo syndrome is relentless and brutal. With children losing ground to this disease every day we have to keep moving forward in efficient and innovative ways. We are hopeful that our PerlQuests will bring us ever closer to help for patients with Sanfilippo syndrome.”
Cara O’Neill, MD.
Chief Science Officer, Cure Sanfilippo Foundation
Chief Science Officer, Cure Sanfilippo Foundation
Our Progress
We are developing nematode and fly disease models and screening compounds for promising leads. Stay tuned!
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