Pegcetacoplan inhibits C3 activation by binding to C3 and its activation fragment C3b, thereby reducing C3 glomerular fragment deposition in patients with C3G and primary IC-MPGN.

Adjunctive corticosteroids not superior to placebo, while adjunctive corneal cross-linking associated with increased scar size.

Sepiapterin is a PAH activator that reduces blood Phe levels by increasing the intracellular concentrations of tetrahydrobiopterin and protecting against PAH enzyme misfolding.