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Preclinical studies have evaluated KD primarily in SOD1 transgenic mouse models, which exhibit progressive motor neuron degeneration, muscle weakness, and mitochondrial dysfunction, thereby recapitulating key features of disease progression [40, 102]

237 A locked nucleic acid (LNA) is a nucleic acid analog with a unique bicyclic structure, where the C4 and O2 atoms are connected by different methylene bridges, forming a stable C3-endo conformation

This includes over-the-counter products, herbal preparations, or medications obtained privately, as many substances can transfer into breast milk

Blood 58(Suppl 1):133a, 1981

Future advances in gene-editing technologies such as CRISPR, precision medicine, and bioprocessing developments could improve the efficacy and accessibility of recombinant enzyme therapy

Error bars indicate the confidence intervals from each flux analysis