Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease-specific assessments.

Sam SalekAudra N BoscoeSarah PiantedosiShayna EganChristopher J EvansTed WellsJennifer CohenRobert J KlaassenRachael GraceMichael Storm

Published in: European journal of haematology (2020)

The PKDD and PKDIA fill a gap in the existing outcomes measurement strategy for PK deficiency. Future work includes psychometric evaluation of these newly developed measures.

Keyphrases

replacement therapy
protein kinase
tyrosine kinase
current status
metabolic syndrome
adipose tissue