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Success will depend on collaborative efforts across basic science, clinical research, and health technology sectors to translate these promising developments into safe, effective, and accessible therapies for the global OA population

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Hohenfellner K, Bergmann C, Fleige T, Janzen N, Burggraf S, Olgemoller B, Gahl WA, Czibere L, Froschauer S, Roschinger W, Vill K, Harms E, Nennstiel U (2019) Molecular based newborn screening in Germany: follow-up for cystinosis