Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency

PURPOSE: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining adequate hemoglobin levels through transfusion...

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Detalles Bibliográficos
Autores principales: Salek, M. S., Ionova, T., Johns, J. R., Oliva, E. N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6373289/
https://www.ncbi.nlm.nih.gov/pubmed/30456713
http://dx.doi.org/10.1007/s11136-018-2025-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6373289/
https://www.ncbi.nlm.nih.gov/pubmed/30456713
http://dx.doi.org/10.1007/s11136-018-2025-y

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