acertx.com
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Acer Therapeutics is a biotech company, headquartered in Cambridge, MA, that develops repurposed and reformulated medicines for the treatment of ultra-orphan diseases with significant unmet medical needs. Acer's lead candidate, ACER-001, is the first pharmaceutical therapy being developed for patients with Maple Syrup Urine Disease (MSUD). MSUD is a devastating genetic disease – an inborn error of amino acid metabolism. Approximately 800 patients suffer from MSUD in the U.S. and 3,000 worldwide. There are no therapeutic options for MSUD and diet is not enough. Despite careful dietary management, the majority of MSUD patients have chronic neurological and social impairment, as well as life-threatening episodes of intoxication. The pivotal clinical program for ACER-001 in MSUD is expected to begin enrolling in 1H 2015. Acer was awarded orphan drug designation by the FDA in August 2014.
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