Clinical features and health-related quality of life in adult patients with mucopolysaccharidosis IVA: the Spanish experience
Abstract Background Mucopolysaccharidosis (MPS) IVA or Morquio A syndrome is a progressive and disabling disease characterized by a deficiency of the enzyme N-acetylgalactosamine-6-sulphate sulphatase. Its clinical presentation is very heterogeneous and poorly understood in adults. The aim of this s...
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| Autores principales: | , , , , , , , , , , , , , , , |
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| Formato: | article |
| Lenguaje: | EN |
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BMC
2021
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| Materias: | |
| Acceso en línea: | https://doaj.org/article/45d102193e154f53a8b2f74d26c527a5 |
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