It has been safely used orally in children for up to 6 months (NatMed 2025)
This could help ensure broader access while maintaining company profitability
These enzymes are involved in pathways that allow for the production of ATP, NADPH, and ribose-5-phosphate which are critical for generating cellular energy and downstream production of amino acids, nucleic acids, and fatty acids (Figure 1)
These modifications include acylation of the peptide backbone with a spacer and a C-18 fatty di-acid chain to lysine at position 26, which mediates strong binding to albumin and potentially results in reduced renal clearance, and an amino-acid substitution in position 8, which results in reduced susceptibility to dipeptidyl peptidase-4 (DPP-4) degradation
As lesions progress, vascular smooth muscle cells (VSMCs), crucial for maintaining structural stability of the arterial wall, also become susceptible to ferroptosis (Zhang et al., 2022)