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Lys251X nonsense mutation (right image) results in a change from lysine at position 251 to a premature STOP codon, causing truncation of the enzyme.

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Only after a review of all diagnostic and therapeutic sleep studies can the optimal treatment approach be determined.

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Figure 2 shows results over time according to treatment for the efficacy variable (change from baseline in “Off” time) that served as the clinical outcome measure at the end of the trial at 12 weeks.

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