Current and emerging treatments and surgical interventions for Morquio A syndrome: a review

Shunji Tomatsu1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USACorrespondence[email protected]

William G Mackenzie1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Mary C Theroux1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Robert W Mason1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Mihir M Thacker1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Thomas H Shaffer1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Adriana M Montañ2 Department of Pediatrics

Daniel Rowan2 Department of Pediatrics

William Sly3 Edward A Doisy Department of Biochemistry and Molecular Biology, Saint Louis University, St Louis, MO, USA

Carlos J Alméciga-Díaz4 Institute for the Study of Inborn Errors of Metabolism, Pontificia Universidad Javeriana, Bogotá DC, Colombia

Luis A Barrera4 Institute for the Study of Inborn Errors of Metabolism, Pontificia Universidad Javeriana, Bogotá DC, Colombia

Yasutsugu Chinen5 Department of Pediatrics, Faculty of Medicine, University of the Ryukyus, Okinawa, Japan

Eriko Yasuda1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Kristen Ruhnke1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA

Yasuyuki Suzuki6 Medical Education Development Center

Tadao Orii7 Department of Pediatrics, Gifu University, Gifu, Japan

Citations (4)

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Chung-Lin Lee, Chih-Kuang Chuang, Huei-Ching Chiu, Ru-Yi Tu, Yun-Ting Lo, Ya-Hui Chang, Shuan-Pei Lin & Hsiang-Yu Lin. (2022) Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome. Drug Design, Development and Therapy 16, pages 143-154.
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Kazuki Sawamoto, Yasuyuki Suzuki, William G. Mackenzie, Mary C. Theroux, Christian Pizarro, Hiromasa Yabe, Kenji E. Orii, Robert W. Mason, Tadao Orii & Shunji Tomatsu. (2016) Current therapies for Morquio A syndrome and their clinical outcomes. Expert Opinion on Orphan Drugs 4:9, pages 941-951.
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Shunji Tomatsu, Kazuki Sawamoto, Carlos J Alméciga-Díaz, Tsutomu Shimada, Michael B Bober, Yasutsugu Chinen, Hiromasa Yabe, Adriana M Montaño, Roberto Giugliani, Francyne Kubaski, Eriko Yasuda, Alexander Rodríguez-López, Angela J Espejo-Mojica, Oscar F Sánchez, Robert W Mason, Luis A Barrera, William G Mackenzie & Tadao Orii. (2015) Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. Drug Design, Development and Therapy 9, pages 1937-1953.
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Shunji Tomatsu, Carlos J Alméciga-Díaz, Hector Barbosa, Adriana M Montaño, Luis A Barrera, Tsutomu Shimada, Eriko Yasuda, William G Mackenzie, Robert W Mason, Yasuyuki Suzuki, Kenji E Orii & Tadao Orii. (2013) Therapies of mucopolysaccharidosis IVA (Morquio A syndrome). Expert Opinion on Orphan Drugs 1:10, pages 805-818.
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