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Rare Disease • 2015 • PLOS One 10(5): e0126025. PMID: 25978616.

Clements D, Dongre A, Krymskaya VP, Johnson SR

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Rare Disease • 2015 • PLoS One 10(3): e0116396. PMID: 25738292.

Koeppen AH, Ramirez RL, Becker AB, Becker AB, Bjork ST, Levi S, Santambrogio P, Parsons PJ, Kruger PC, Yang KX, Feustel PJ, Mazurkiewicz JE

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Rare Disease • 2015 • The Journal of Clinical Investigation 125(9): 3449-3460. PMID: 26241054.

Wooton-Kee CR, Jain AK, Wagner M, Grusak MA, Finegold MJ, Lutsenko S, Moore DD

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