Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study
尼曼匹克病 C 型的临床疾病进展和生物标志物：一项前瞻性队列研究
期刊:Orphanet Journal of Rare Diseases
影响因子:3.4
doi:10.1186/s13023-020-01616-0
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
