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Retrograde transport of toxins accross the endoplasmic reticulum membrane

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Individuals with ARTHS present with developmental delay, cognitive impairment (100% penetrance), delay or absence of speech (100%), facial dysmorphism (85%), feeding difficulties (78%), and neonatal hypotonia (76%) in a study of 76 individuals with KAT6A mutations (Kennedy et al, 2019)

Patients may prefer troches for convenience, ease of self-administration, and steady systemic effects that support overall tissue regeneration