Rare Disease Report

Gene Therapy for Lipoprotein Lipase Deficiency Coming to North America

JUNE 12, 2015



Deya Corzo, MD, FAAP, FACMG, vice president of US medical affairs at uniQure talks about the clinical trial the company has planned in North America to study the safety and efficacy of Glybera (alipogene tiparvovec) for the treatment of patients with lipoprotein lipase deficiency (also known as familial chylomicronemia syndrome). The gene therapy is currently approved for treatment in Europe.

For more information, visit www.uniqure.com or www.lplda.org

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