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Article
Open AccessEteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study
Duchenne muscular dystrophy (DMD) is characterized by rapid functional decline. Current available treatment options aim to delay disease progression or stabilize physical function. To aid in healthcare provide...
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Article
Open AccessA Qualitative Study to Develop and Evaluate the Content Validity of the Vitiligo Patient Priority Outcome (ViPPO) Measures
Vitiligo can be associated with a psychological burden, stigmatization and impaired quality of life. Tools to assess the impact of vitiligo exist; however, none were developed in line with the FDA’s patient-re...
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Article
Open AccessMeaningful Changes in What Matters to Individuals with Vitiligo: Content Validity and Meaningful Change Thresholds of the Vitiligo Area Scoring Index (VASI)
This study explored patients’ and dermatologists’ priority outcomes for treatment to address, clinical outcome assessments (COA) for use in vitiligo clinical trials, and perceptions of within-patient meaningfu...