Selective Neutral Sphingomyelinase-2 Inhibition Therapy

h-var-f354525002

**Molecular Mechanism and Rationale** The pathophysiological foundation of this therapeutic approach centers on the dysregulated activity of neutral sphingomyelinase-2 (nSMase2), encoded by the SMPD3 gene, which catalyzes the hydrolysis of sphingomyelin to ceramide and phosphocholine at the plasma membrane. Unlike its lysosomal counterpart acid sphingomyelinase (ASMase/SMPD1), nSMase2 operates op