Pancreatitis reduced by nearly 50 percent after gene therapy to treat lipoprotein lipase deficiency - (Science Daily via NewsPoints Desk)

  • Research data published in the journal Human Gene Therapy reveal that uniQure's gene therapy Glybera reduced the severity and frequency of pancreatitis, Science Daily reported Tuesday.

  • In the study, researchers from Chiesi Farmaceutici, Erasmus Medical Centre, uniQure and other institutions examined the safety and efficacy of a single dose of Glybera over a six-year period, observing no cases of severe pancreatitis and a single intensive care unit admission for a disease lipoprotein lipase deficiency-related abdominal event.

  • "As gene therapy moves forward into the mainstream of medicine, it will be critical to define the evidence of its benefit to patients with specific diseases," commented University of Massachusetts Medical School deputy chancellor Terence Flotte.

  • "This work with alipogene tiparvovec provides an evidence-based guideline for its use in patients with LPLD going forward," Flotte continued.

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