Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey

BACKGROUND: Morquio A syndrome (or mucopolysaccharidosis IVa) is an ultra-rare multi-organ disease, resulting in significantly impaired functional capacity, mobility and quality of life (QoL). METHODS: This patient-reported outcomes survey evaluated the global burden of Morquio A among adults (≥18 y...

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Detalles Bibliográficos
Autores principales: Hendriksz, Christian J, Lavery, Christine, Coker, Mahmut, Ucar, Sema Kalkan, Jain, Mohit, Bell, Lisa, Lampe, Christina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4016149/
https://www.ncbi.nlm.nih.gov/pubmed/24602160
http://dx.doi.org/10.1186/1750-1172-9-32

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4016149/
https://www.ncbi.nlm.nih.gov/pubmed/24602160
http://dx.doi.org/10.1186/1750-1172-9-32

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