Bridging the gap in lysosomal disorders drug development: A call for endpoint innovation and economic foresight

Molecular Genetics and Metabolism 2026

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Authors: Kathleen Coolidge, Daniel Lewi, Gerald F. Cox, Michael B. Nichol, Justin Hopkin, Femida hussein Gwadry-Sridhar

DOI: 10.1016/j.ymgme.2025.109373

Cited by: 0

Fields of Study & Subjects

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