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Neuronal ceroid lipofuscinoses type 7 (CLN7): a case series reporting cross sectional and retrospective clinical data to evaluate validity of standardized tools to assess disease progression, quality of life, and adaptive skills.
Kayani S, BordesEdgar V, Lowden A, Nettesheim ER, Dahshi H, Messahel S, Minassian BA, Greenberg BM. Kayani S, et al. Orphanet J Rare Dis. 2024 Dec 19;19(1):468. doi: 10.1186/s13023-024-03448-8. Orphanet J Rare Dis. 2024. PMID: 39702211 Free PMC article.
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