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...

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Bibliografske podrobnosti
izdano v:Drug Des Devel Ther
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: Dove Medical Press 2015
Teme:
Review
Online dostop: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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