Epidemiology of Mucopolysaccharidoses Update von Çelik, Betul, Tomatsu, Saori C, Tomatsu, Shunji, Khan, Shaukat A

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Newborn screening of mucopolysaccharidoses: past, present, and future von Arunkumar, Nivethitha, Langan, Thomas J, Stapleton, Molly, Kubaski, Francyne, Mason, Robert W, Singh, Rajendra, Kobayashi, Hironori, Yamaguchi, Seiji, Suzuki, Yasuyuki, Orii, Kenji, Orii, Tadao, Fukao, Toshiyuki, Tomatsu, Shunji

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Glycosaminoglycans detection methods: Applications of mass spectrometry von Kubaski, Francyne, Osago, Harumi, Mason, Robert W, Yamaguchi, Seiji, Kobayashi, Hironori, Tsuchiya, Mikako, Orii, Tadao, Tomatsu, Shunji

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Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments von Sawamoto, Kazuki, Stapleton, Molly, Alméciga-Díaz, Carlos J., Espejo-Mojica, Angela J., Losada, Juan Camilo, Suarez, Diego A., Tomatsu, Shunji

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Effect of enzyme replacement therapy on the growth of patients with Morquio A von Doherty, Caitlin, Stapleton, Molly, Piechnik, Matthew, Mason, Robert W, Mackenzie, William G, Yamaguchi, Seiji, Kobayashi, Hironori, Suzuki, Yasuyuki, Tomatsu, Shunji

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Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I von Gomez-Ospina, Natalia, Scharenberg, Samantha G., Mostrel, Nathalie, Bak, Rasmus O., Mantri, Sruthi, Quadros, Rolen M., Gurumurthy, Channabasavaiah B., Lee, Ciaran, Bao, Gang, Suarez, Carlos J., Khan, Shaukat, Sawamoto, Kazuki, Tomatsu, Shunji, Raj, Nitin, Attardi, Laura D., Aurelian, Laure, Porteus, Matthew H.

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Evading the AAV Immune Response in Mucopolysaccharidoses von Piechnik, Matthew, Sawamoto, Kazuki, Ohnishi, Hidenori, Kawamoto, Norio, Ago, Yasuhiko, Tomatsu, Shunji

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Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future von Chen, Hui Hsuan, Sawamoto, Kazuki, Mason, Robert W, Kobayashi, Hironori, Yamaguchi, Seiji, Suzuki, Yasuyuki, Orii, Kenji, Orii, Tadao, Tomatsu, Shunji

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Hearing Loss in Mucopolysaccharidoses: Current Knowledge and Future Directions von Wolfberg, Jeremy, Chintalapati, Keerthana, Tomatsu, Shunji, Nagao, Kyoko

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Newborn screening and diagnosis of mucopolysaccharidoses von Tomatsu, Shunji, Fujii, Tadashi, Fukushi, Masaru, Oguma, Toshihiro, Shimada, Tsutomu, Maeda, Miho, Kida, Kazuhiro, Shibata, Yuniko, Futatsumori, Hideyuki, Montaño, Adriana M., Mason, Robert W., Yamaguchi, Seiji, Suzuki, Yasuyuki, Orii, Tadao

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UPLC-MS/MS detection of disaccharides derived from glycosaminoglycans as biomarkers of mucopolysaccharidoses von Auray-Blais, Christiane, Lavoie, Pamela, Tomatsu, Shunji, Valayannopoulos, Vassili, Mitchell, John J., Raiman, Julian, Beaudoin, Maxime, Maranda, Bruno, Clarke, Joe T.R.

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Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice von Rintz, Estera, Celik, Betul, Fnu, Nidhi, Herreño-Pachón, Angélica María, Khan, Shaukat, Benincore-Flórez, Eliana, Tomatsu, Shunji

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Surgical Reconstruction for Severe Tracheal Obstruction in Morquio A Syndrome von Pizarro, Christian, MD, Davies, Ryan R., MD, Theroux, Mary, MD, Spurrier, Ellen A., MD, Averill, Lauren W., MD, Tomatsu, Shunji, MD

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Obstructive airway in Morquio A syndrome, the past, the present and the future von Tomatsu, Shunji, Averill, Lauren W., Sawamoto, Kazuki, Mackenzie, William G., Bober, Michael B., Pizarro, Christian, Goff, Christopher J., Xie, Li, Orii, Tadao, Theroux, Mary

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Newborn screening for mucopolysaccharidoses: a pilot study of measurement of glycosaminoglycans by tandem mass spectrometry von Kubaski, Francyne, Mason, Robert W., Nakatomi, Akiko, Shintaku, Haruo, Xie, Li, van Vlies, Naomi N., Church, Heather, Giugliani, Roberto, Kobayashi, Hironori, Yamaguchi, Seiji, Suzuki, Yasuyuki, Orii, Tadao, Fukao, Toshiyuki, Montaño, Adriana M., Tomatsu, Shunji

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Therapies for the bone in mucopolysaccharidoses von Tomatsu, Shunji, Alméciga-Díaz, Carlos J., Montaño, Adriana M., Yabe, Hiromasa, Tanaka, Akemi, Dung, Vu Chi, Giugliani, Roberto, Kubaski, Francyne, Mason, Robert W., Yasuda, Eriko, Sawamoto, Kazuki, Mackenzie, William, Suzuki, Yasuyuki, Orii, Kenji E., Barrera, Luis A., Sly, William S., Orii, Tadao

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Tracheal narrowing in children and adults with mucopolysaccharidosis type IVA: evaluation with computed tomography angiography von Averill, Lauren W., Kecskemethy, Heidi H., Theroux, Mary C., Mackenzie, William G., Pizarro, Christian, Bober, Michael B., Ditro, Colleen P., Tomatsu, Shunji

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Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice von Leal, Andrés Felipe, Celik, Betul, Fnu, Nidhi, Khan, Shaukat, Tomatsu, Shunji, Alméciga-Díaz, Carlos Javier

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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases von Rintz, Estera, Higuchi, Takashi, Kobayashi, Hiroshi, Galileo, Deni S., Wegrzyn, Grzegorz, Tomatsu, Shunji

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Biomarkers in patients with mucopolysaccharidosis type II and IV von Fujitsuka, Honoka, Sawamoto, Kazuki, Peracha, Hira, Mason, Robert W., Mackenzie, William, Kobayashi, Hironori, Yamaguchi, Seiji, Suzuki, Yasuyuki, Orii, Kenji, Orii, Tadao, Fukao, Toshiyuki, Tomatsu, Shunji

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