6c. Management



Substrate reduction therapy: This acts differently from enzyme replacement therapy. Rather than giving the body back the enzyme that is lacking, we are using a peptide that will block the step above that enzyme so that substrate does not accumulate, so that substrate that is supposed to be degraded is not made, so the body has less of it accumulating and the body has to work less hard to degrade what there is. There are a couple that are available right now, Zavesca for Gaucher and other LSDs, we also have Cerdelga, which is another Gaucher Disease substrate reduction therapy.





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