Investigating the real-world experience of patients with Niemann-Pick disease type C (NPC) and their carers: Preliminary results

Molecular Genetics and Metabolism 2026

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Authors: Krista Casazza, Toni Mathieson, Sandra Cowie, Rebecca C. Knibb, Jackson Pountney, Solomon Mbua, Justin Hopkin, Shaun Bolton

DOI: 10.1016/j.ymgme.2025.109356

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Genetic Neurodegenerative Diseases