A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa

A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa

Abstract Background Standardized assessments for dystrophic epidermolysis bullosa (DEB) are needed. This prospective, multicenter, 4-week, observational study was designed to evaluate DEB assessments for suitability as clinical trial endpoints. Methods Patients with confirmed DEB diagnosis and ≥ 5 m...

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Bibliographic Details
Main Authors: Amy S. Paller, Elena Pope, Dan Rudin, Anna Malyala, Deborah Ramsdell, Ramsey Johnson, Hal Landy, Dedee F. Murrell, the DEB Investigators
Format: Article
Language:English
Published: BMC 2022-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Dystrophic epidermolysis bullosa
Clinician-assessed outcomes
Quality of life
Patient-reported outcomes
Disease severity
Outcome measures
Online Access:https://doi.org/10.1186/s13023-022-02461-z

Internet

https://doi.org/10.1186/s13023-022-02461-z

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