6 Result(s)
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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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Open AccessDevelopment of a Conceptual Model for the Patient Experience of Immunoglobulin A Nephropathy (IgAN): A Qualitative Literature Review
Immunoglobulin A nephropathy (IgAN) is a kidney disorder that can lead to progressive kidney disease. Currently, there lacks a comprehensive overview of the symptoms and impacts experienced by those living wit...
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Open AccessDevelopment of a Conceptual Model for the Patient Experience of Focal Segmental Glomerulosclerosis (FSGS): A Qualitative Targeted Literature Review
Focal segmental glomerulosclerosis (FSGS) is a leading cause of kidney disease and can progress to end stage kidney disease (ESKD). An overview of symptoms and impacts of the disease experienced will help info...
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Open AccessKey measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials
This study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population wi...
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Open AccessDevelopment of Clinician-Reported Outcome (ClinRO) and Patient-Reported Outcome (PRO) Measures for Eyebrow, Eyelash and Nail Assessment in Alopecia Areata
Eyebrow and eyelash hair loss and nail damage—in addition to scalp hair loss—are important signs/symptoms of alopecia areata (AA) to patients and deserve assessment in AA clinical trials.
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Open AccessCognitive Impairment Associated with Schizophrenia: A Review of the Humanistic Burden
Nearly every individual with schizophrenia is affected by cognitive decline. The aim of this literature review was to: (a) describe the humanistic burden of cognitive impairment associated with schizophrenia (...
