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This approach facilitates personalized interventions such as DGAT1 inhibitors for obese patients exhibiting astrocytic lipid droplet accumulation [278], or NAD + boosters for elderly patients with impaired ketone utilization, as exemplified in the SPAN trial [47, 48, 255]

Early initiation of treatment after the onset of vision loss and a longer treatment duration appears to be associated with better odds of improving or stabilising vision

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Accurate and early detection of treatable IEMs through NBS can significantly reduce disease burden and healthcare costs