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Evolving therapies in neuronopathic LSDs: opportunities and challenges

Deepa S Rajan1, Maria L Escolar2

  • 1Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.

Metabolic Brain Disease
|April 20, 2022
PubMed

Keywords:
Enzyme replacement therapyGene therapyHematopoietic stem cell therapyLysosomal storage disorders (LSDs)NeurodegenerationSubstrate reduction therapy

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