Damage at the cellular and organ levels in LSDs: possibility for prevention/reversibility with ERT

摘要

Reversal of pathology at the cellular, tissue and organ levels is one of the aims of treatment in patients with lysosomal storage diseases (LSDs). However, there are many factors that can impact upon the ability of exogeneous enzyme to clear cellular inclusions. The efficacy of the infused enzyme, for example, may be affected not only by the properties of the enzyme itself, but also by the degree of existing pathology and other factors that are specific to the disease or affected tissue. The potential of enzyme replacement therapy (ERT) to reverse or prevent the development of disease was discussed in the symposium session 'Damage at the cellular and organ level in LSDs: possibility for prevention/reversibility with ERT'.