substrate reduction therapy

What are the Challenges Associated with SRT?

Despite its benefits, SRT faces several challenges. One of the main issues is specificity. Ensuring that the SRT targets only the problematic substrate without affecting other metabolic pathways is crucial. Another challenge is the potential for side effects, as reducing substrate levels can sometimes lead to unintended consequences. Additionally, the effectiveness of SRT can vary among individuals due to genetic differences and the severity of the enzyme deficiency.

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