Addressing the Multisystemic Impacts of Nephropathic Cystinosis in an Adult by Jeanine R. Jarnes, Rebekah S. Palmer, Chester B. Whitley

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Dose-intensive therapy (DIT) for infantile Pompe disease: A pilot study by Jeanine R. Jarnes, Nishitha R. Pillai, Alia Ahmed, Sofia Shrestha, Molly Stark, Chester B. Whitley

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Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series by Nishitha R. Pillai, Ning Liu, Xiyuan Li, Xiqi Li, Rebecca Ahrens-Nicklas, Laura Adang, Julie B. Eisengart, Grace Bronken, Ashish Gupta, Troy C. Lund, Chester B. Whitley, Sarah H. Elsea, Paul J. Orchard

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