Barely Significant
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Development and evaluation of RhizoQOL, a quality-of-life caregiver-reported survey for rhizomelic chondrodysplasia punctata, a rare peroxisomal disorder.

Orphanet J Rare Dis · 2025 · PMC11956500 · PMID 40165314

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an overall trendno p-value reported
In addition, while a statistically significant change may not be feasible, an overall trend of improvement on the RhizoQOL would provide strong support for the clinical meaningfulness of changes observed in primary or secondary endpoints.

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