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

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

Abstract 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 dire...

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Bibliographic Details
Main Authors: Alexandra Morrison, Esmee Oussoren, Tabea Friedel, Jordi Cruz, Nalan Yilmaz
Format: Article
Language:English
Published: BMC 2019-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Mucopolysaccharidosis type VII
MPS VII
Sly disease
Lysosomal storage disorder
Burden of illness
Diagnostic delay
Online Access:http://link.springer.com/article/10.1186/s13023-019-1233-z

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http://link.springer.com/article/10.1186/s13023-019-1233-z

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