Treatment In Lysosomal Storage Disorders

What this episode covers

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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Frequently Asked Questions

How long is this episode of JIMD Podcasts?

This episode is 24 minutes long.

When was this JIMD Podcasts episode published?

This episode was published on June 25, 2021.

What is this episode about?

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...

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