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Small-Molecule Precursors Restore CoQ10 and Reverse Neurological Decline in HPDL Deficiency

The Nature paper introduces a translational framework for testing CoQ10 headgroup intermediates in clinical trials

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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.