Glybera - Biotech, Pharma and Life Science Channel
uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency. LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million. For further information on LPLD visit www.lpldeficiency.com.
Glybera has been tested in three interventional clinical studies conducted in the Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein.
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uniQure's Glybera® First Gene Therapy Approved by European Commission
AMSTERDAM, The Netherlands, November 2, 2012 /PRNewswire/ -- Glybera becomes the first gene therapy approved by regulatory authorities in the Western world First medication approved for...
Glybera becomes the first gene therapy approved by regulatory authorities in the Western world First medication approved for patients with rare metabolic disorder Lipoprotein Lipase Defici...
Pending EC decision: Glybera , alipogene tiparvovec, Opinion date: 19-Jul-2012
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Clinical Trials
This trial is designed to expand the currently available data on the safety and efficacy of alipogene tiparvovec treatment in lipoprotein lipase deficiency (LPLD) and to further the unders...
PubMed Articles
The catalytic activity of the Family 4 glycosidase LplD protein, whose active site motif is CHEV, is unknown despite its crystal structure having been determined in 2008. Here we identify that activit...
AIM: To evaluate the technical feasibility, safety, and oncological outcomes of laparoscopic extended lateral pelvic lymph node dissection (LPLD) following total mesorectal excision (TME) in patients...
We describe the 2-year follow-up of an open-label trial (CT-AMT-011-01) of AAV1-LPL(S447X) gene therapy for lipoprotein lipase (LPL) deficiency (LPLD), an orphan disease associated with chylomicronemi...
