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.