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Article
Open AccessDevelopment of Novel Patient-Reported Outcome (PRO) and Observer-Reported Outcome (ObsRO) Instruments in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA): ViSIO-PRO and ViSIO-ObsRO
Retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) are rare inherited retinal degenerative disorders resulting in visual impairments and impacts on patients’ vision-dependent activities of daily li...
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Article
Open AccessQualitative exploration of the visual function impairments and impacts on vision-dependent activities of daily living in Retinitis Pigmentosa and Leber Congenital Amaurosis: content validation of the ViSIO-PRO and ViSIO-ObsRO measures
Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) are rare inherited retinal degenerative disorders. The associated visual impairments have significant impacts on patients’ vision-dependent activi...
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Article
Open AccessPsychometric Validation of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis
Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) are rare inherited retinal degenerative disorders. The Visual Symptom and Impact Outcomes patient-reported outcome (ViSIO-PRO) and observer-report...
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Article
Open AccessComparison of Literature Review, Social Media Listening, and Qualitative Interview Research Methods in Generating Patient-Reported Symptom and Functional Impact Concepts of Presbyopia
To compare the insights obtained about the experience of individuals with presbyopia (age-related impaired near vision) across three different sources of qualitative data: a structured targeted literature revi...
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Article
Open AccessDevelopment and content validation of a self-completed, electronic Pediatric Asthma Symptom Diary
Childhood asthma is an important unmet need. To date, patient-reported outcome measures (PROMs) for children with asthma have used a combination of caregiver or proxy-reported and self-reported measures. No co...
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Article
Open AccessEffect of indacaterol/glycopyrronium on ventilation and perfusion in COPD: a randomized trial
The long-acting β2-agonist/long-acting muscarinic antagonist combination indacaterol/glycopyrronium (IND/GLY) elicits bronchodilation, improves symptoms, and reduces exacerbations in COPD. Magnetic resonance imag...
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Article
Open AccessUnderstanding the visual function symptoms and associated functional impacts of phakic presbyopia
Presbyopia is defined as the age-related deterioration in the ability to focus on close objects, causing difficulty with near vision tasks. The study aim was to understand the lived experience of phakic presby...
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Article
Open AccessEvaluation of the content validity of patient-reported outcome (PRO) instruments developed for use with individuals with phakic presbyopia, including the Near Activity Visual Questionnaire-presbyopia (NAVQ-P) and the near vision correction independence (NVCI) instrument
Presbyopia is the age-related deterioration in the ability to focus on close objects. In order to develop a patient-reported outcome (PRO) instrument to assess near vision functioning, the Near Activity Visual...
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Article
Open AccessMeasuring What Matters for Children: A Systematic Review of Frequently Used Pediatric Generic PRO Instruments
To provide an assessment of the quality of the most frequently used self-reported, generic patient-reported outcome measures (PROMs) that measure health-related quality of life (HRQoL) in children against the ...
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Article
Open AccessImpact of baseline symptoms and health status on COPD exacerbations in the FLAME study
COPD is a heterogeneous disease and patients may respond differently to therapies depending on baseline symptom burden.
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Article
Open AccessGathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice
We illustrate our experience of gathering patient insights on the most patient-relevant symptoms in chronic obstructive pulmonary disease (COPD) via a structured and systematic approach towards ‘patient-centri...
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Article
Open AccessIndacaterol/glycopyrronium versus salmeterol/fluticasone in the prevention of clinically important deterioration in COPD: results from the FLAME study
Chronic obstructive pulmonary disease (COPD) is a progressive disease and a composite endpoint could be an indicator of treatment effect on disease worsening. This post-hoc analysis assessed whether indacatero...
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Article
Open AccessEfficacy and safety of direct switch to indacaterol/glycopyrronium in patients with moderate COPD: the CRYSTAL open-label randomised trial
Dual bronchodilation combining a long-acting β2-agonist (LABA) and a long-acting muscarinic antagonist (LAMA) is the preferred choice of treatment recommended by the Global Initiative for Chronic Obstructive Lung...
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Chapter
Anti-IgE Agents
There is little debate over the essential role of IgE in allergic reactions. During an allergic response, CD4+ T lymphocytes of the Th2 phenotype stimulate allergen-specific B cells to produce IgE molecules via t...
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Chapter
The development of iralukast: A member of the Novartis series of cysteinyl leukotriene antagonists
The development of specific antagonists of cysteinyl leukotrienes (Cys-LTs) has provided therapeutic agents for use in diseases of the respiratory tract by antagonizing the bronchoconstrictive, mucus secretory...
