Records containing: Nephropathic Cystinosis
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CLINICAL_TRIALS study record update · 2026-04-27An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase
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CLINICAL_TRIALS study record update · 2025-11-10An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase
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CLINICAL_TRIALS study record update · 2025-05-29Coordination of Rare Diseases at Sanford
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CLINICAL_TRIALS study record update · 2025-03-03A European, Multicenter, Prospective Clinical Study to Evaluate Cysteamine Toxicity on Human Osteoclasts. The CYSTEA-BONE Clinical Study.
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CLINICAL_TRIALS study record update · 2021-11-29Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis
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CLINICAL_TRIALS study record update · 2014-03-12Cure Cystinosis International Registry
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CLINICAL_TRIALS study record update · 2013-10-09Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults