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Researchers collaborate to seek FDA approval for drug treatment for rare disease

Author: Gene Stowe and Marissa Gebhard

Norbert Wiech with students

University of Notre Dame alumnus Norbert Wiech founded Lysomics LLC to manage the clinical development needed to bring to market a promising new treatment for people with Niemann-Pick Type C (NPC) disease. FDA support is being sought for early clinical exploration of an approved drug to fight this rare disease that has no cure or treatment.

Lysomics is based on the work of Notre Dame professors of chemistry and biochemistry Olaf Wiest and Paul Helquist, and Frederick Maxfield at Cornell University’s Weill Medical College, to find treatments for NPC. NPC disease is a rare, fatal neurodegenerative disease that primarily strikes children before and during adolescence. Read More