Overview

• Nature study shows 4-HMA and 4-HB supplementation bypasses HPDL deficiency to restore CoQ10 synthesis.
• Oral dosing of these precursors rescued over 90% of HPDL-deficient mice from paralysis, allowing normal development.
• Under FDA expanded access, an eight-year-old boy regained walking and running ability within two months of 4-HB treatment.
• NYU Langone’s integrated research infrastructure and U.S. regulatory approvals enabled rapid translation from mouse studies to patient care.
• Researchers are planning larger clinical trials to determine optimal dosing, treatment windows and to secure regulatory and licensing clearances.