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Antoine Labbé, Christophe Baudouin, Georges Deschênes, Chantal Loirat, Marina Charbit, Geneviève Guest, Patrick Niaudet
OBJECTIVE: To establish the safety and efficacy of a new gel formulation of cysteamine hydrochloride (CH) eye drops, for the treatment of corneal complications of nephropathic cystinosis. DESIGN: Open label dose response clinical trial. PARTICIPANTS: Eight patients with infantile nephropathic cystinosis including 4 children, 3 adolescents, and 1 adult (mean age at inclusion, 12.1 ± 4.6 years) treated with CH 0.1% eye drops. INTERVENTION: Patients were treated, in both eyes, with the control CH 0...
March 2014: Molecular Genetics and Metabolism
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