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.

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

Models to study basic and applied aspects of lysosomal storage disorders - ScienceDirect

Advancements in Niemann-Pick Disease Type C Treatments

Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis: Molecular Therapy - Methods & Clinical Development

Frontiers CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges

Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation

Niemann–Pick disease type C1 (NPC1) lysosomal lipid storage disease

New gene therapy targets brain disorders

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

Promising gene replacement therapy moves forward

Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease

Niemann-Pick Disease, Type C: Diagnosis, Management and Disease-Targeted Therapies in Development - ScienceDirect