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38 ensaios clínicos encontrados, 11 ativos.
An Australian standard of care for Niemann-Pick disease type C.
Comparison of neonatal systemic and intracerebroventricular AAV9 gene therapy delivery demonstrating improved behavioral and phenotypic outcomes in a mouse model of Niemann-Pick disease, type C1.
Case Report: Interleukin-23 blockade achieves sustained remission in Niemann-Pick type C-associated Crohn's disease refractory to anti-tumor necrosis factor therapy.
Suspected Niemann-Pick disease type B with sea-blue histiocytosis after splenectomy: A rare case report.
Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.