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Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)
Paul Gissen, Nicola Specchio, Andrew Olaye and 14 more
Orphanet Journal of Rare Diseases · 2021
doi:10.1186/s13023-021-01829-x
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