Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science
Grabowski GA, Mistry PK. Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science. Molecular Genetics And Metabolism 2022, 137: 81-91. PMID: 35933791, DOI: 10.1016/j.ymgme.2022.07.014.Peer-Reviewed Original Research