With Regard to Treatment of Inborn Errors of Metabolism (IEM)

question 21

Multiple Choice

With regard to treatment of inborn errors of metabolism (IEM) , which of the following statements, if any, is false?

• A) IEMs are all single gene disorders that have been studied for many decades, leading to the development of successful treatment in all cases.
• B) IEMs can be treated by augmentation therapy, treatment to inhibit positively harmful effects, or by prevention therapy.
• C) Treatment for some individual IEMs can involve both augmentation therapy plus treatment to inhibit positively harmful effects.
• D) Treatment of some IEMs involves artificially forcing an increase in a minor metabolic pathway to counteract a build-up in a toxic metabolite produced by a metabolic block in a major metabolic pathway.

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