Can selective acid sphingomyelinase modulators achieve therapeutic ceramide reduction without triggering Niemann-Pick-like pathology?

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Description

The debate proposed partial SMPD1 inhibition as promising but didn’t address the safety margin between therapeutic benefit and lysosomal dysfunction. This knowledge gap is critical for clinical translation given the known risks of complete sphingomyelinase inhibition.

Source: Debate session sess_SDA-2026-04-01-gap-lipid-rafts-2026-04-01 (Analysis: SDA-2026-04-01-gap-lipid-rafts-2026-04-01)

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