Miglustat for treatment of Niemann-Pick C disease: a randomised
Por um escritor misterioso
Descrição
Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts
Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
IJMS, Free Full-Text
Unbiased yeast screens identify cellular pathways affected in Niemann–Pick disease type C
Ocular motor biomarkers in Niemann-Pick disease type C: A prospective cross-sectional multicontinental study in 72 patients
Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect
Miglustat in patients with Niemann-Pick disease Type C (NP-C): A
Recommendations for the detection and diagnosis of Niemann-Pick disease type C
Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling - ScienceDirect
Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases
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