Conclusion
PK deficiency
K DEFICIENCY, DISEASE
The results presented here provide evidence of validity and reliability for the first disease-specific PROs for the assessment of the symptoms and disease impact of PK deficiency in patients within a clinical trial setting, the PKDD and PKDIA, and support the PKDD and PKDIA as valid, reliable, and responsive tools to measure and evaluate changes in disease impact in clinical trials for adults with PK deficiency.