Advanced search
Publication Cover
Drug Design, Development and Therapy Volume 9, 2015 - Issue
Submit an article Journal homepage
130
Views
54
CrossRef citations to date
0
Altmetric
Review

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome

Shunji Tomatsu1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA;2 Department of Pediatrics, Gifu University, Gifu, JapanCorrespondence[email protected]
,
Kazuki Sawamoto1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA
,
Carlos J Alméciga-Díaz3 Institute for the Study of Inborn Errors of Metabolism, School of Sciences, Pontificia Universidad Javeriana, Bogotá, Colombia
,
Tsutomu Shimada1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA
,
Michael B Bober1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA
,
Yasutsugu Chinen4 Department of Pediatrics, Faculty of Medicine, University of the Ryukyus, Okinawa, Japan
,
Hiromasa Yabe5 Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara, Japan
,
Adriana M Montaño6 Department of Pediatrics, Saint Louis University, St Louis, MO, USA
,
Roberto Giugliani7 Medical Genetics Service/HCPA and Department of Genetics/UFRGS, Porto Alegre, Brazil
,
Francyne Kubaski1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA;8 Department of Biological Sciences, University of Delaware, Newark, DE, USA
,
Eriko Yasuda1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA
,
Alexander Rodríguez-López3 Institute for the Study of Inborn Errors of Metabolism, School of Sciences, Pontificia Universidad Javeriana, Bogotá, Colombia
,
Angela J Espejo-Mojica3 Institute for the Study of Inborn Errors of Metabolism, School of Sciences, Pontificia Universidad Javeriana, Bogotá, Colombia
,
Oscar F Sánchez9 School of Chemical Engineering, Purdue University, West Lafayette, IN, USA
,
Robert W Mason1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA
,
Luis A Barrera3 Institute for the Study of Inborn Errors of Metabolism, School of Sciences, Pontificia Universidad Javeriana, Bogotá, Colombia
,
William G Mackenzie1 Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, USA
&
Tadao Orii2 Department of Pediatrics, Gifu University, Gifu, Japan
 show all
Pages 1937-1953 | Published online: 01 Apr 2015

Citations (54)

Keep up to date with the latest research on this topic with citation updates for this article.

Subscribe to citation updates

Read on this site (5)

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.
Read now
Fabiano Poswar, Guilherme Baldo & Roberto Giugliani. (2017) Phase I and II clinical trials for the mucopolysaccharidoses. Expert Opinion on Investigational Drugs 26:12, pages 1331-1340.
Read now
Christian J. Hendriksz. (2016) Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome). Expert Review of Clinical Pharmacology 9:12, pages 1521-1532.
Read now
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.
Read now
Shunji Tomatsu, Kazuki Sawamoto, Tsutomu Shimada, Michael B Bober, Francyne Kubaski, Eriko Yasuda, Robert W Mason, Shaukat Khan, Carlos J Alméciga-Díaz, Luis A Barrera, William G Mackenzie & Tadao Orii. (2015) Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations. Expert Opinion on Orphan Drugs 3:11, pages 1279-1290.
Read now

Articles from other publishers (49)

Sulaiman Mohammed Alnasser. (2022) Stem cell challenge in cancer progression, oncology and therapy. Gene 840, pages 146748.
Crossref
Yuhua Qu, Hao Liu, Likun Wei, Shushan Nie, Wenjiao Ding, Sha Liu, Haiyan Liu & Hua Jiang. (2022) The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation From Different Donors in Recipients With Mucopolysaccharidosis. Frontiers in Pediatrics 10.
Crossref
Lorena Diaz-Ordoñez, Estephania Candelo, Katherine Silva-Cuero, Wilmar Saldarriaga, Lenka Murgašová, Martin Magner & Harry Pachajoa. (2022) Hearing Loss in Patients With Morquio Syndrome: Protocol for a Scoping Review. JMIR Research Protocols 11:6, pages e32986.
Crossref
Young Bae Sohn, Curtis Rogers, Jennifer Stallworth, Jessica A. Cooley Coleman, Laura Buch, Erin Jozwiak, Jo Ann Johnson, Tim Wood, Paul Harmatz, Laura Pollard & Raymond J. Louie. (2022) RNA analysis of the GALNS transcript reveals novel pathogenic mechanisms associated with Morquio syndrome A. Molecular Genetics and Metabolism Reports 31, pages 100875.
Crossref
Michael Flanagan, Isha Pathak, Qi Gan, Linda Winter, Ryan Emnet, Salem Akel & Adriana M. Montaño. (2021) Umbilical mesenchymal stem cell-derived extracellular vesicles as enzyme delivery vehicle to treat Morquio A fibroblasts. Stem Cell Research & Therapy 12:1.
Crossref
Hui Chen, Shaukat Khan, Betul Celik, Yasuyuki Suzuki, Yasuhiko Ago & Shunji Tomatsu. (2021) Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy. Molecular Genetics & Genomic Medicine 9:11.
Crossref
José V. Álvarez, Susana B. Bravo, María Pilar Chantada-Vázquez, Sofía Barbosa-Gouveia, Cristóbal Colón, Olalla López-Suarez, Shunji Tomatsu, Francisco J. Otero-Espinar & María L. Couce. (2021) Plasma Proteomic Analysis in Morquio A Disease. International Journal of Molecular Sciences 22:11, pages 6165.
Crossref
Ruchi Shah, Cynthia Amador, Kati Tormanen, Sean Ghiam, Mehrnoosh Saghizadeh, Vaithi Arumugaswami, Ashok Kumar, Andrei A. Kramerov & Alexander V. Ljubimov. (2021) Systemic diseases and the cornea. Experimental Eye Research 204, pages 108455.
Crossref
Eric Goldman, Angela Vu, Kelly Dietz & Stefani N Thomas. (2021) A 9-Month-Old with Skeletal Abnormalities and a Consanguineous Sibling with Mucopolysaccharidosis IVA: The Role of Urinary Glycosaminoglycan Testing in Disease Diagnosis and Treatment Monitoring. Clinical Medicine Insights: Case Reports 14, pages 117954762199940.
Crossref
Víctor J. Álvarez, Susana B. Bravo, Maria Pilar Chantada-Vazquez, Cristóbal Colón, María J. De Castro, Montserrat Morales, Isidro Vitoria, Shunji Tomatsu, Francisco J. Otero-Espinar & María L. Couce. (2020) Characterization of New Proteomic Biomarker Candidates in Mucopolysaccharidosis Type IVA. International Journal of Molecular Sciences 22:1, pages 226.
Crossref
Zhirui Jiang, Sharon Byers, Margret L. Casal & Lachlan J. Smith. (2020) Failures of Endochondral Ossification in the Mucopolysaccharidoses. Current Osteoporosis Reports 18:6, pages 759-773.
Crossref
Kazuki Sawamoto, Subha Karumuthil-Melethil, Shaukat Khan, Molly Stapleton, Joseph T. Bruder, Olivier Danos & Shunji Tomatsu. (2020) Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model. Molecular Therapy - Methods & Clinical Development 18, pages 50-61.
Crossref
Jeremy Wolfberg, Keerthana Chintalapati, Shunji Tomatsu & Kyoko Nagao. (2020) Hearing Loss in Mucopolysaccharidoses: Current Knowledge and Future Directions. Diagnostics 10:8, pages 554.
Crossref
Rossella Parini & Federica Deodato. (2020) Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations. International Journal of Molecular Sciences 21:8, pages 2975.
Crossref
Esteban A. Gonzalez, Fernanda Visioli, Gabriela Pasqualim, Carolina F. M. Souza, Diane R. Marinho, Roberto Giugliani, Ursula Matte & Guilherme Baldo. (2020) Progressive eye pathology in mucopolysaccharidosis type I mice and effects of enzyme replacement therapy. Clinical & Experimental Ophthalmology 48:3, pages 334-342.
Crossref
Kazuki Sawamoto, José Álvarez González, Matthew Piechnik, Francisco Otero, Maria Couce, Yasuyuki Suzuki & Shunji Tomatsu. (2020) Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management. International Journal of Molecular Sciences 21:4, pages 1517.
Crossref
Angela C. Sosa, Barbara Kariuki, Qi Gan, Alan P. Knutsen, Clifford J. Bellone, Miguel A. Guzmán, Luis A. Barrera, Shunji Tomatsu, Anil K. Chauhan, Eric Armbrecht & Adriana M. Montaño. (2020) Oral immunotherapy tolerizes mice to enzyme replacement therapy for Morquio A syndrome. Journal of Clinical Investigation 130:3, pages 1288-1300.
Crossref
Mehmet Umut Akyol, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz & Maurizio Scarpa. (2019) Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. Orphanet Journal of Rare Diseases 14:1.
Crossref
Hui Hsuan Chen, Kazuki Sawamoto, Robert W. Mason, Hironori Kobayashi, Seiji Yamaguchi, Yasuyuki Suzuki, Kenji Orii, Tadao Orii & Shunji Tomatsu. (2019) Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future. Journal of Human Genetics 64:11, pages 1153-1171.
Crossref
J. Víctor Álvarez, Susana B. Bravo, María García-Vence, María J. De Castro, Asteria Luzardo, Cristóbal Colón, Shunji Tomatsu, Francisco J. Otero-Espinar & María L. Couce. (2019) Proteomic Analysis in Morquio A Cells Treated with Immobilized Enzymatic Replacement Therapy on Nanostructured Lipid Systems. International Journal of Molecular Sciences 20:18, pages 4610.
Crossref
Nato D. Vashakmadze, Leyla S. Namazova-Baranova, Natalia V. Zhurkova, Ekaterina Yu. Zakharova, Svetlana V. Mikhaylova & Grigory V. Revunenkov. (2019) Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation Results in Patients with Hurler Syndrome: Clinical Cases. Current Pediatrics 18:3, pages 196-202.
Crossref
Kazuki Sawamoto & Shunji Tomatsu. (2019) Development of Substrate Degradation Enzyme Therapy for Mucopolysaccharidosis IVA Murine Model. International Journal of Molecular Sciences 20:17, pages 4139.
Crossref
Alexander Rodríguez-López, Luisa N. Pimentel-Vera, Angela J. Espejo-Mojica, Annelies Van Hecke, Petra Tiels, Shunji Tomatsu, Nico Callewaert & Carlos J. Alméciga-Díaz. (2019) Characterization of Human Recombinant N-Acetylgalactosamine-6-Sulfate Sulfatase Produced in Pichia pastoris as Potential Enzyme for Mucopolysaccharidosis IVA Treatment. Journal of Pharmaceutical Sciences 108:8, pages 2534-2541.
Crossref
Caitlin Doherty, Molly Stapleton, Matthew Piechnik, Robert W. Mason, William G. Mackenzie, Seiji Yamaguchi, Hironori Kobayashi, Yasuyuki Suzuki & Shunji Tomatsu. (2019) Effect of enzyme replacement therapy on the growth of patients with Morquio A. Journal of Human Genetics 64:7, pages 625-635.
Crossref
Madeleine Taylor, Shaukat Khan, Molly Stapleton, Jianmin Wang, Jing Chen, Robert Wynn, Hiromasa Yabe, Yasutsugu Chinen, Jaap Jan Boelens, Robert W. Mason, Francyne Kubaski, Dafne D.G. Horovitz, Anneliese L. Barth, Marta Serafini, Maria Ester Bernardo, Hironori Kobayashi, Kenji E. Orii, Yasuyuki Suzuki, Tadao Orii & Shunji Tomatsu. (2019) Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future. Biology of Blood and Marrow Transplantation 25:7, pages e226-e246.
Crossref
Kazuki Sawamoto, Molly Stapleton, Carlos J. Alméciga-Díaz, Angela J. Espejo-Mojica, Juan Camilo Losada, Diego A. Suarez & Shunji Tomatsu. (2019) Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments. Drugs 79:10, pages 1103-1134.
Crossref
Akitaka Shibata, Takenori Yoshikawa, Sumiko Makita, Hiroyuki Takama, Takeshi Arakawa & Masashi Akiyama. (2019) Urticarial vasculitis and subcutaneous nodules in the extremities seen in a patient with mucopolysaccharidosis II after hematopoietic stem cell therapy. The Journal of Dermatology 46:2, pages e63-e64.
Crossref
Nicole Williams, Alessandro Narducci, Deborah M Eastwood, Maureen Cleary & Dominic Thompson. (2018) An Evidence-based Approach to the Management of Children With Morquio A Syndrome Presenting With Craniocervical Pathology. Spine 43:24, pages E1443-E1453.
Crossref
Anna Caciotti, Rodolfo Tonin, Matthew Mort, David N. Cooper, Serena Gasperini, Miriam Rigoldi, Rossella Parini, Federica Deodato, Roberta Taurisano, Michelina Sibilio, Giancarlo Parenti, Renzo Guerrini & Amelia Morrone. (2018) Mis-splicing of the GALNS gene resulting from deep intronic mutations as a cause of Morquio a disease. BMC Medical Genetics 19:1.
Crossref
Rossella Parini & Andrea Biondi. (2018) The new frame for Mucopolysaccharidoses. Italian Journal of Pediatrics 44:S2.
Crossref
Carlos J. Alméciga-Díaz, Adriana M. Montaño, Luis A. Barrera & Shunji Tomatsu. (2018) Tailoring the AAV2 capsid vector for bone-targeting. Pediatric Research 84:4, pages 545-551.
Crossref
Hira Peracha, Kazuki Sawamoto, Lauren Averill, Heidi Kecskemethy, Mary Theroux, Mihir Thacker, Kyoko Nagao, Christian Pizarro, William Mackenzie, Hironori Kobayashi, Seiji Yamaguchi, Yasuyuki Suzuki, Kenji Orii, Tadao Orii, Toshiyuki Fukao & Shunji Tomatsu. (2018) Molecular genetics and metabolism, special edition: Diagnosis, diagnosis and prognosis of Mucopolysaccharidosis IVA. Molecular Genetics and Metabolism 125:1-2, pages 18-37.
Crossref
Kazuki Sawamoto, Hui-Hsuan Chen, Carlos J. Alméciga-Díaz, Robert W. Mason & Shunji Tomatsu. (2018) Gene therapy for Mucopolysaccharidoses. Molecular Genetics and Metabolism 123:2, pages 59-68.
Crossref
Yasemin Tanyildizi, Seyfullah Gökce, Federico Marini, Anna K Mayer, Stefanie Kirschner, Julia B. Hennermann & Marc A. Brockmann. (2017) Vessel shape alterations of the vertebrobasilar arteries in Mucopolysaccharidosis type IVa (Morquio A) patients. European Journal of Radiology 93, pages 128-133.
Crossref
Bruna Donida, Desirèe P. Marchetti, Carlos Eduardo Diaz Jacques, Graziela Ribas, Marion Deon, Paula Manini, Helen Tais da Rosa, Dinara Jaqueline Moura, Jenifer Saffi, Roberto Giugliani & Carmen Regla Vargas. (2017) Oxidative profile exhibited by Mucopolysaccharidosis type IVA patients at diagnosis: Increased keratan urinary levels. Molecular Genetics and Metabolism Reports 11, pages 46-53.
Crossref
Nabiha Saklayen, Marinus Huber, Marinna Madrid, Valeria Nuzzo, Daryl I. Vulis, Weilu Shen, Jeffery Nelson, Arthur A. McClelland, Alexander Heisterkamp & Eric Mazur. (2017) Intracellular Delivery Using Nanosecond-Laser Excitation of Large-Area Plasmonic Substrates. ACS Nano 11:4, pages 3671-3680.
Crossref
Tamer Ahmed EL-Sobky, Alaa El-Haddad, Ezzat Elsobky, Solaf M. Elsayed & Hossam Moussa Sakr. (2017) Reversal of skeletal radiographic pathology in a case of malignant infantile osteopetrosis following hematopoietic stem cell transplantation. The Egyptian Journal of Radiology and Nuclear Medicine 48:1, pages 237-243.
Crossref
Douglas Colmenares-Bonilla & Nayeli Esquitin-Garduño. (2017) Diagnosis of Morquio-A patients in Mexico: How far are we from prompt diagnosis?. Intractable & Rare Diseases Research 6:2, pages 119-123.
Crossref
Shaukat Khan, Carlos J. Alméciga-Díaz, Kazuki Sawamoto, William G. Mackenzie, Mary C. Theroux, Christian Pizarro, Robert W. Mason, Tadao Orii & Shunji Tomatsu. (2017) Mucopolysaccharidosis IVA and glycosaminoglycans. Molecular Genetics and Metabolism 120:1-2, pages 78-95.
Crossref
Alexander Rodríguez-López, Carlos J. Alméciga-Díaz, Jhonnathan Sánchez, Jefferson Moreno, Laura Beltran, Dennis Díaz, Andrea Pardo, Aura María Ramírez, Angela J. Espejo-Mojica, Luisa Pimentel & Luis A. Barrera. (2016) Recombinant human N-acetylgalactosamine-6-sulfate sulfatase (GALNS) produced in the methylotrophic yeast Pichia pastoris. Scientific Reports 6:1.
Crossref
Jorge Luis Suarez-Guerrero, Pedro José Iván Gómez Higuera, Juan Sebastian Arias Flórez & Gustavo Adolfo Contreras-García. (2016) Mucopolisacaridosis: características clínicas, diagnóstico y de manejo. Revista Chilena de Pediatría 87:4, pages 295-304.
Crossref
Eriko Yasuda, Yasuyuki Suzuki, Tsutomu Shimada, Kazuki Sawamoto, William G. Mackenzie, Mary C. Theroux, Christian Pizarro, Li Xie, Freeman Miller, Tariq Rahman, Heidi H. Kecskemethy, Kyoko Nagao, Thierry Morlet, Thomas H. Shaffer, Yasutsugu Chinen, Hiromasa Yabe, Akemi Tanaka, Haruo Shintaku, Kenji E. Orii, Koji O. Orii, Robert W. Mason, Adriana M. Montaño, Toshiyuki Fukao, Tadao Orii & Shunji Tomatsu. (2016) Activity of daily living for Morquio A syndrome. Molecular Genetics and Metabolism 118:2, pages 111-122.
Crossref
Calogera M. Simonaro, Shunji Tomatsu, Tracy Sikora, Francyne Kubaski, Michael Frohbergh, Johana M. Guevara, Raymond Y. Wang, Moin Vera, Jennifer L. Kang, Lachlan J. Smith, Edward H. Schuchman & Mark E. Haskins. (2016) Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs. PLOS ONE 11:4, pages e0153136.
Crossref
S. B. RobertsR. DrydenA. I. Tsirikos. (2016) Thoracolumbar kyphosis in patients with mucopolysaccharidoses. The Bone & Joint Journal 98-B:2, pages 229-237.
Crossref
Hiromasa Yabe, Akemi Tanaka, Yasutsugu Chinen, Shunichi Kato, Kazuki Sawamoto, Eriko Yasuda, Haruo Shintaku, Yasuyuki Suzuki, Tadao Orii & Shunji Tomatsu. (2016) Hematopoietic stem cell transplantation for Morquio A syndrome. Molecular Genetics and Metabolism 117:2, pages 84-94.
Crossref
Simon Jones & Frits A. Wijburg. 2016. Inborn Metabolic Diseases. Inborn Metabolic Diseases 577 590 .
Sun H. Peck, Philip J.M. O'Donnell, Jennifer L. Kang, Neil R. Malhotra, George R. Dodge, Maurizio Pacifici, Eileen M. Shore, Mark E. Haskins & Lachlan J. Smith. (2015) Delayed hypertrophic differentiation of epiphyseal chondrocytes contributes to failed secondary ossification in mucopolysaccharidosis VII dogs. Molecular Genetics and Metabolism 116:3, pages 195-203.
Crossref
Ángela J. Espejo-Mojica, Carlos J. Alméciga-Díaz, Alexander Rodríguez, Ángela Mosquera, Dennis Díaz, Laura Beltrán, Sergio Díaz, Natalia Pimentel, Jefferson Moreno, Jhonnathan Sánchez, Oscar F. Sánchez, Henry Córdoba, Raúl A. Poutou-Piñales & Luis A. Barrera. (2015) Human recombinant lysosomal enzymes produced in microorganisms. Molecular Genetics and Metabolism 116:1-2, pages 13-23.
Crossref
Chunxiao Qi, Xiaojun Yan, Chenyu Huang, Alexander Melerzanov & Yanan Du. (2015) Biomaterials as carrier, barrier and reactor for cell-based regenerative medicine. Protein & Cell 6:9, pages 638-653.
Crossref

Related research

People also read lists articles that other readers of this article have read.

Recommended articles lists articles that we recommend and is powered by our AI driven recommendation engine.

Cited by lists all citing articles based on Crossref citations.
Articles with the Crossref icon will open in a new tab.