Harvard Catalyst Profiles

Contact, publication, and social network information about Harvard faculty and fellows.

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

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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Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.