ArticleOphthalmology and therapy2025
Cystadrops
Article in Ophthalmology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
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0 citing papers in PubMed.
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionCystinosis is a multisystemic disease manifesting in the eyes initially as asymptomatic corneal cystine crystals and later with photophobia and serious visual impairment. Systemic effects of cystinosis arise from multiple cellular dysfunctions, causing early presymptomatic effects and progressive complications. Corneal crystals are observed across all layers of the cornea from infancy, and crystal accumulation during childhood is rapid. Early treatment is imperative, but in Europe, at the time of this study, no topical therapy was licensed for patients < 2 years of age.
methodsThis study was a paediatric investigation plan approved by the European Medicines Agency to assess the safety profile and efficacy of Cystadrops
resultsAll primary endpoints were met. Any adverse events were mild and did not prevent treatment continuation. Corneal cystine crystal and photophobia scores declined or remained constant in all patients at a stage in life when an increase might be expected.
conclusionAlthough the rarity of the disease renders large studies on infants impracticable, there is no indication that the safety and efficacy profile of Cystadrops CLINICAL TRIAL REGISTRY: Clinical Trials No: 2018-002984-24.
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