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This trial will study the safety, tolerability, and efficacy of CERC-801 in patients with PGM1-CDG using daily therapeutic doses of CERC-801 in approximately ten patients.
Lead Product(s): Alpha-D-Galactose
Therapeutic Area: Genetic Disease Product Name: CERC-801
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Cerecor
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration November 17, 2020