Treatment In Lysosomal Storage Disorders

25/06/2021 24 min
Treatment In Lysosomal Storage Disorders

Listen "Treatment In Lysosomal Storage Disorders"

Episode Synopsis

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

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