Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients

BACKGROUND: Research about pediatric patients’ perspective on mucopolysaccharidosis type VI (MPS VI) and its impact on daily life is limited. We aimed to identify the disease concepts of interest that most impact function and day-to-day life of pediatric patients with MPS VI, and to consider clinica...

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Detalles Bibliográficos
Autores principales: Leiro, Beth, Phillips, Dawn, Duiker, Melanie, Harmatz, Paul, Charles, Sharon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8638175/
https://www.ncbi.nlm.nih.gov/pubmed/34857033
http://dx.doi.org/10.1186/s13023-021-02113-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8638175/
https://www.ncbi.nlm.nih.gov/pubmed/34857033
http://dx.doi.org/10.1186/s13023-021-02113-8

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