Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study
Eugen Mengel, Bruno Bembi, Mireia Del Toro, Federica Deodato, Matthias Gautschi, Stephanie Grunewald, Sabine Grønborg, Bénédicte Héron, Esther M Maier, Agathe Roubertie, Saikat Santra, Anna Tylki-Szymanska, Simon Day, Tara Symonds, Stacie Hudgens, Marc C Patterson, Christina Guldberg, Linda Ingemann, Nikolaj H T Petersen, Thomas KirkegaardChristine Í Dali
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