Current therapies for Morquio A syndrome and their clinical outcomes

INTRODUCTION: Morquio A syndrome is characterized by a unique skeletal dysplasia, leading to short neck and trunk, pectus carinatum, laxity of joints, kyphoscoliosis, and tracheal obstruction. Cervical spinal cord compression/inability, a restrictive and obstructive airway, and/or bone deformity and...

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Publicat a:Expert Opin Orphan Drugs
Autors principals: Sawamoto, Kazuki, Suzuki, Yasuyuki, Mackenzie, William G., Theroux, Mary C., Pizarro, Christian, Yabe, Hiromasa, Orii, Kenji E., Mason, Robert W., Orii, Tadao, Tomatsu, Shunji
Format: Artigo
Idioma:Inglês
Publicat: 2016
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5312776/
https://ncbi.nlm.nih.gov/pubmed/28217429
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2016.1214572
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