Treating mucopolysaccharidosis type IIIC with small molecule therapy that stabilizes lysosomal function and reduces inflammation

Molecular Genetics and Metabolism 2026

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Authors: Rafael A. Badell‐Grau, Tara Shammas, Veenita Khare, Stéphanie Cherqui, Johannes C. M. Schlachetzki

DOI: 10.1016/j.ymgme.2025.109322

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Biomedical Research and Pathophysiology