Treating Niemann-Pick Disease Type C

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Niemann–Pick disease - Wikipedia

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

About NPD - International Niemann-Pick Disease Alliance (INPDA)

Niemann-Pick Disease, Types C1 (D)

A treatment increases survival by 50% in mice with Niemann-Pick type C, a rare disease that affects brain and liver

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Niemann-Pick Disease disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

Anesthetic consideration of Niemann-Pick Disease type C

Niemann-Pick disease type C-presenting as persistent neonatal jaundice: a rare case report

Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin

Niemann-Pick Disease Type C

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology