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University of Washington, Seattle Shotelersuk V, Larson D, Anikster Y, McDowell G, Lemons R, Bernardini I, Guo J, Thoene J, Gahl WA (1998) CTNS mutations in an American-based population of cystinosis patients

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Larger randomized trials are still needed to understand who is most likely to benefit, how durable the effect is, and why some people may respond more strongly than others