-
1
Ablation of lipocalin-2 reduces neuroinflammation in a mouse model of Krabbe disease
Published 2024-12-01Subjects: “…Lysosomal storage disorder…”
Get full text
Article -
2
Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial
Published 2025-01-01Subjects: Get full text
Article -
3
Advancing CNS Therapeutics: Enhancing Neurological Disorders with Nanoparticle-Based Gene and Enzyme Replacement Therapies
Published 2025-02-01Subjects: Get full text
Article -
4
Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Published 2025-01-01Subjects: Get full text
Article