Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach
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DOI: 10.1186/s13023-022-02208-w
Abstract: Background 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… read more here.
Keywords: mps iiia; age; severity stages; qualitatively derived ... See more keywords