Validation of a novel patient reported tool to assess the impact of treatment in erythropoietic protoporphyria: the EPP-QoL

• Autores principales: G. Biolcatti (Autor), S. Hanneken (Autor), E. I. Minder (Autor), N. J. Neumann (Autor), J. H. P. Wilson (Autor), P. J. Wolgen (Autor), D. J. Wright (Autor), A. J. Lloyd (Autor)
• Formato: Libro
• Publicado: SpringerOpen, 2021-08-01T00:00:00Z.
• Materias: article
• Acceso en línea: Connect to this object online.

Abstract Background A novel treatment has been developed for erythropoietic protoporphyria (EPP) (a rare condition that leaves patients highly sensitive to light). To fully understand the burden of EPP and the benefit of treatment, a novel patient reported outcome (PRO) measure was developed called...