Treating Niemann-Pick C lysosomal storage: approved and emerging approaches

Trends Mol Med. 2024 Dec 12:S1471-4914(24)00313-7. doi: 10.1016/j.molmed.2024.11.011. Online ahead of print.

Authors

Andrés D Klein¹, Emily R Eden², Silvana Zanlungo³

Affiliations

¹ Centro de Genética y Genómica, Facultad de Medicina, Clínica Alemana Universidad del Desarrollo, Santiago, Chile. Electronic address: [email protected].
² UCL Institute of Ophthalmology, London, UK.
³ Department of Gastroenterology, Faculty of Medicine, Pontificia Universidad Católica de Chile, Santiago, Chile.

PMID: 39672736
DOI: 10.1016/j.molmed.2024.11.011

No abstract available

Keywords: Niemann-Pick type C; lysosomal storage disorders; neurodegeneration; rare disease; therapeutics.