Experimental treatment for Niemann-Pick disease appears safe

An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested

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Experimental treatment for Niemann-Pick disease appears safe

IJMS, Free Full-Text

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

Identification of Novel Biomarkers for Niemann–Pick Disease Using Gene Expression Analysis of Acid Sphingomyelinase Knockout Mice: Molecular Therapy

Niemann-Pick disease Information

The Neuropsychiatry of Niemann-Pick Type C Disease in Adulthood

Niemann-Pick Disease Concise Medical Knowledge

INPDA International Niemann-Pick Disease Alliance

Parents of twins with Niemann-Pick find a treatment