Bone Abstracts

Objectives: Lysosomal diseases (LDs) are a heterogenous group of 40+ genetic disorders that can affect virtually all organs and systems, including the skeletal system. They are often caused by the loss of an enzyme critical for the breakdown of macromolecules in the lysosome, leading to accumulation of these substrates and subsequent lysosomal dysfunction. Enzyme replacement therapy (ERT) is the primary treatment option for many LDs, but several issues hinder the efficacy of t...