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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, EDVISO™ (celiprolol), is the first pharmaceutical therapy seeking approval for patients with vascular-type Ehlers-Danlos syndrome (vEDS). Ehlers-Danlos Syndrome is a group of hereditary disorders of connective tissue with no ethnic predisposition. vEDS is a subtype characterized by severe arterial dissections, ruptures and early death. Complications are rare in childhood, but affect 25% of patients before the age of 20, and 80% by the age of 40. Median age of death is estimated to be around 50 years. Patients are diagnosed by clinical symptoms and confirmed by presence of mutations in the COL3A1 gene. There are approximately 2,000 documented patients, but prevalence could be as high as 5,000 patients in the U.S. (Pepin 2014). There are no specific pharmacological treatments for vEDS and medical intervention centers on symptomatic treatment, prophylactic measures and genetic counseling. Acer was awarded orphan drug designation by the FDA in January 2015. Acer`s second 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. Acer was awarded orphan drug designation by the FDA in August 2014.
Simcere Pharmaceuticals is a New York, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Velocity Clinical Research, headquartered in Durham, NC, is a leading integrated site organization for clinical trials, offering dedicated site capabilities to help biopharmaceutical and contract research organization customers find the right patients for their studies. The company has 18 sites across 12 US States. We place the care of the patient at the heart of everything we do. With over 35 years of experience running sites and more than 5500 studies completed, Velocity has refined its patient recruitment strategies while maintaining a focus on delivering timely and reliable data quality.
J M Smith Corporation is a Spartanburg, SC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.