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PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation.

Tan L, Narayan SB, Chen J, Meyers GD, Bennett MJ. PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation. J Inherit Metab Dis. 2011 Apr; 34(2):443-7.

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