Pathway to diagnosis and burden of illness in mucopolysaccharidosis type VII – a European caregiver survey

BACKGROUND: Mucopolysaccharidosis type VII (Sly disease, MPS VII), is an ultra-rare, multi-symptom disease with variable clinical presentations which can present challenges with diagnosis, management and care. We believe this survey is the first to explore the patient experience through direct quest...

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Detalles Bibliográficos
Autores principales: Morrison, Alexandra, Oussoren, Esmee, Friedel, Tabea, Cruz, Jordi, Yilmaz, Nalan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6854616/
https://www.ncbi.nlm.nih.gov/pubmed/31727109
http://dx.doi.org/10.1186/s13023-019-1233-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6854616/
https://www.ncbi.nlm.nih.gov/pubmed/31727109
http://dx.doi.org/10.1186/s13023-019-1233-z

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