Searchable abstracts of presentations at key conferences on calcified tissues

ba0007oc10 | (1) | ICCBH2019

Next-generation antibody-guided enzyme replacement therapy for lysosomal storage diseases

Baik Andrew , Aaron Nina , Birnbaum Matthew , Calafati Philip , Schoenherr Christopher , Economides Aris , Cygnar Katherine

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...