6d. Management



The chaperone therapy is another modality. It’s also another peptide that will bind to the enzyme that is left over - noting that there are different severities of LSDs depending on how much residual enzyme activity there is - certain people don’t have 0% enzyme activity, they have 1%, they have 2%, they have some enzymes that is functional but that enzyme is not enough to do all of the work. The chaperone binds to that enzyme and tries to correct some of the issues that makes that enzyme defective. It may be that enzyme is not going to where it’s supposed to go, or maybe it’s not being folded the right way so the chaperone is going to remediate that, and the enzyme will become more functional. The good thing about both the substrate reduction therapy and the chaperone therapy is that both cross the blood-brain barrier, so for those neurological LSDs where enzyme replacement therapy is unable to do that maybe in the future we could combine both a substrate reduction therapy and an enzyme replacement therapy to target every organ in the body.





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