Treating lysosomal storage disorders: What have we learnt?

Abstract excerpt

The first enzyme replacement therapy (ERT) for a lysosomal storage disorder (LSD) was approved in 1991 and we now have more than 25 years of experience of treating patients with type 1 Gaucher disease. Because of the remarkable success of this therapy, enormous effort and resource has gone into developing other ERTs, for Gaucher (where three different enzyme preparations have now been approved) and for other...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Autoimmune and Inflammatory Disorders Research
• Share to Lysosomal Storage Disorders Research
• Share to Trypanosoma species research and implications