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Access and affordability constraints limit the population that can benefit from current therapies

L-Carnitine reduced exercise-induced catabolism (muscle breakdown), reduced free-radical generation, and minimized muscle tissue disruption compared to the placebo

The occluded aqueous cavity housing the Na + -binding site is unusual, and we hypothesise that Na + binding and ordering of solvent within this cavity may stabilise the N-terminal domain and contribute to transporter gating

The 6-month and 12-month cohorts were treated independently, so that any given individual could be included in either cohort, or in both, provided they had data available at the relevant time points