Mapping Lysosomal Storage Disorders with Neurological Features by Cellular Pathways: Towards Precision Medicine

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Review reveals precision medicine approaches in lysosomal storage disorders, highlighting neurological impacts and therapeutic options.

Neurodegeneration is a prominent feature in lysosomal storage disorders affecting children's development.
Genetics and cell biology are foundational in understanding the diverse clinical phenotypes seen across disorders.
A cellular-pathway-oriented framework is proposed for better classification and therapeutic targeting.
This approach supports the development of personalized strategies like gene therapy and enzyme replacement.

Makridou et al. (2025) studied this question.

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