Below are the most recent publications written about "Enzyme Replacement Therapy" by people in Profiles.
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Leal AF, Benincore-Fl?rez E, Rintz E, Herre?o-Pach?n AM, Celik B, Ago Y, Alm?ciga-D?az CJ, Tomatsu S. Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets. Int J Mol Sci. 2022 Dec 28; 24(1).
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Mitchell JJ, Burton BK, Bober MB, Campeau PM, Cohen S, Dosenovic S, Ellaway C, Bhattacharya K, Guffon N, Hinds D, Lail A, Lin SP, Magner M, Raiman J, Schwartz-Sagi L, Stepien KM. Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa. Mol Genet Metab. 2022 Sep-Oct; 137(1-2):164-172.
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Chen H, Khan S, Celik B, Suzuki Y, Ago Y, Tomatsu S. Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy. Mol Genet Genomic Med. 2021 11; 9(11):e1806.
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?lvarez JV, Bravo SB, Chantada-V?zquez MP, Barbosa-Gouveia S, Col?n C, L?pez-Suarez O, Tomatsu S, Otero-Espinar FJ, Couce ML. Plasma Proteomic Analysis in Morquio A Disease. Int J Mol Sci. 2021 Jun 07; 22(11).
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?lvarez VJ, Bravo SB, Chantada-Vazquez MP, Col?n C, De Castro MJ, Morales M, Vitoria I, Tomatsu S, Otero-Espinar FJ, Couce ML. Characterization of New Proteomic Biomarker Candidates in Mucopolysaccharidosis Type IVA. Int J Mol Sci. 2020 Dec 28; 22(1).
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Ficicioglu C, Matalon DR, Luongo N, Menello C, Kornafel T, Degnan AJ. Diagnostic journey and impact of enzyme replacement therapy for mucopolysaccharidosis IVA: a sibling control study. Orphanet J Rare Dis. 2020 11 30; 15(1):336.
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Sawamoto K, ?lvarez JV, Herre?o AM, Otero-Espinar FJ, Couce ML, Alm?ciga-D?az CJ, Tomatsu S. Bone-Specific Drug Delivery for Osteoporosis and Rare Skeletal Disorders. Curr Osteoporos Rep. 2020 10; 18(5):515-525.
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Khan SA, Tomatsu SC. Mucolipidoses Overview: Past, Present, and Future. Int J Mol Sci. 2020 Sep 17; 21(18).
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Santi L, De Ponti G, Dina G, Pievani A, Corsi A, Riminucci M, Khan S, Sawamoto K, Antolini L, Gregori S, Annoni A, Biondi A, Quattrini A, Tomatsu S, Serafini M. Neonatal combination therapy improves some of the clinical manifestations in the Mucopolysaccharidosis type I murine model. Mol Genet Metab. 2020 07; 130(3):197-208.
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Sosa AC, Kariuki B, Gan Q, Knutsen AP, Bellone CJ, Guzm?n MA, Barrera LA, Tomatsu S, Chauhan AK, Armbrecht E, Monta?o AM. Oral immunotherapy tolerizes mice to enzyme replacement therapy for Morquio A syndrome. J Clin Invest. 2020 03 02; 130(3):1288-1300.