Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for M...
Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach
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Publisher
England: BioMed Central Ltd
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Language
English
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Publisher
England: BioMed Central Ltd
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MPS IIIA is a rare, degenerative pediatric genetic disease characterized by symptoms impacting cognition, mobility and behavior; the mean age of death is around 15 years of age. Currently, there are no approved therapies for MPS IIIA.
A two-year, multi-center, prospective, descriptive cohort study was conducted to document the natural history co...
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Full title
Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach
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TN_cdi_doaj_primary_oai_doaj_org_article_13f5855d754b4424a5c8cc19369ada71
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_13f5855d754b4424a5c8cc19369ada71
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-022-02208-w
