Treatment In Lysosomal Storage Disorders

Professor Robin Lachmann of the Charles Dent Metabolic Unit discusses the science behind Enzyme Replacement Therapy and Gene Therapy and explains why these do, and sometimes do not, work in Lysosomal Storage Disorders. Professor Lachmann also discusses the progress towards establishing adult metabolic services in the UK and elsewhere. Treating lysosomal storage disorders: What have we learnt? Robin H. Lachmann https://doi.org/10.1002/jimd.12131 Education and training in adult metabolic medicine: Results of an international survey Annalisa Sechi, et al https://doi.org/10.1002/jmd2.12044