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...

Descripción completa

Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2015
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4389814/
https://www.ncbi.nlm.nih.gov/pubmed/25897204
http://dx.doi.org/10.2147/DDDT.S68562

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4389814/
https://www.ncbi.nlm.nih.gov/pubmed/25897204
http://dx.doi.org/10.2147/DDDT.S68562

Ejemplares similares