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Diagnostic workup and management of patients with isolated methylmalonic acidurias in European metabolic centres

DSIP clinical & mechanistic profile DSIP is a naturally occurring nonapeptide discovered in 1974 that promotes delta wave sleep, reduces stress, and modulates multiple neurotransmitter systems including GABA and endogenous opioids

and truly translating cutting-edge technologies such as PROTAC-ADCs and siRNA-ADCs into clinical practice and real-world cohorts to form an interdisciplinary closed-loop validation