Gene therapy shows promise for treating Niemann-Pick disease type C1

NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.

Advancements in Niemann-Pick Disease Type C Treatments

Niemann–Pick type C disease as proof‐of‐concept for intelligent biomarker panel selection in neurometabolic disorders - Papandreou - 2022 - Developmental Medicine & Child Neurology - Wiley Online Library

Niemann-Pick disease type C as a neurovisceral disease. Schematic

Identification of cerebral spinal fluid protein biomarkers in Niemann-Pick disease, type C1, Biomarker Research

Brain gene therapy with Trojan horse lipid nanoparticles: Trends in Molecular Medicine

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

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Family races to raise money for research no one will fund - The Boston Globe

Niemann–Pick Disease Type C

Drug Delivery System for Niemann Pick Type C1 Disease Discovered

Gene therapy shows promise for treating Niemann-Pick disease type C1