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

Patients with mucopolysaccharidosis IVA (MPS IVA) can present with systemic skeletal dysplasia, leading to a need for multiple orthopedic surgical procedures, and often become wheelchair bound in their teenage years. Studies on patients with MPS IVA treated by enzyme replacement therapy (ERT) showed...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Drug Des Devel Ther
Prif Awduron: Tomatsu, Shunji, Sawamoto, Kazuki, Alméciga-Díaz, Carlos J, Shimada, Tsutomu, Bober, Michael B, Chinen, Yasutsugu, Yabe, Hiromasa, Montaño, Adriana M, Giugliani, Roberto, Kubaski, Francyne, Yasuda, Eriko, Rodríguez-López, Alexander, Espejo-Mojica, Angela J, Sánchez, Oscar F, Mason, Robert W, Barrera, Luis A, Mackenzie, William G, Orii, Tadao
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Dove Medical Press 2015
Pynciau:
Review
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4389814/
https://ncbi.nlm.nih.gov/pubmed/25897204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S68562
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