Therapies of mucopolysaccharidosis IVA (Morquio A syndrome)

INTRODUCTION: Morquio A syndrome (mucopolysaccharidosis type IVA, MPS IVA) is one of the lysosomal storage diseases and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme leads to accumulation of glycosaminoglycans (GAGs), keratan sulfate (KS)...

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Pubblicato in:Expert Opin Orphan Drugs
Autori principali: Tomatsu, Shunji, Alméciga-Díaz, Carlos J, Barbosa, Hector, Montaño, Adriana M, Barrera, Luis A, Shimada, Tsutomu, Yasuda, Eriko, Mackenzie, William G, Mason, Robert W, Suzuki, Yasuyuki, Orii, Kenji E, Orii, Tadao
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4238063/
https://ncbi.nlm.nih.gov/pubmed/25419501
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2013.846853
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