Treatment Options for Niemann-Pick Type C

Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush Medical College in Chicago, describes dif

Niemann-Pick disease type C

Combination therapy a potential strategy for treating Niemann-Pick disease

Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy - Daozhan Yu, Manju Swaroop, Mengqiao Wang, Ulrich Baxa, Rongze Yang, Yiping Yan, Turhan

Niemann–Pick Disease Type C

Niemann-Pick disease type C: Video & Anatomy

Treatment Options for Niemann-Pick Type C

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

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Speaker Series - Niemann-Pick Disease Type C

Valley family fights for drug to save sick child

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts