Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale
Patterson, Marc C ; Lloyd-Price, Lucy ; Guldberg, Christina ; Doll, Helen ; Burbridge, Claire ; Chladek, Michael ; íDali, Christine ; Mengel, Eugen ; Symonds, Tara
Orphanet journal of rare diseases, 2021-02, Vol.16 (1), p.79-79, Article 79 [Periódico revisado por pares]England: BioMed Central Ltd
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