Retrophin Begins Dosing Patients in Trial of RE-024

Retrophin has begun dosing patients in a clinical trial assessing the safety and efficacy of RE-024 to treat pantothenate kinase-associated neurodegeneration (PKAN), a rare, genetic neurological disorder. “The dosing of the first patient in the phase 3 FORT Study is an important milestone for the RE-024 program,” said Bill Rote, head of research and development for Retrophin, “as it continues our progress towards developing a treatment with the potential to make a meaningful difference for the PKAN community.”

RE-024 is a novel, investigational, small molecule replacement therapy that aims to restore levels of Coenzyme A (CoA), a naturally occurring molecule that is essential in many cellular functions and is decreased in individuals with PKAN.

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