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Despite significant individual differences, Xpd TTD/TTD mice were found to run 1.37 ± 0.54 km/day on average, compared to 9.37 ± 1.1 km/day seen for wildtype mice, arguing they are indeed less mobile (Fig

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Furthermore, limited diagnostic capabilities, difficulty in identifying eligible patients, and variability in disease progression collectively contribute to low enrollment rates, prolonged study timelines, and compromised data integrity in rare disease trials, ultimately resulting in higher failure rates compared to non-rare disease trials 5,6