Thursday, 8 November 2012

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Netherlands biotech company UniQure have announced that Glybera has become the first gene therapy approved by regulatory authorities in the Western world.

It is the first medication approved for patients with rare metabolic disorder Lipoprotein Lipase Deficiency and commercial roll-out to begin second half of 2013. The company that has taken this product over from AMT state that it now validates uniQure’s unique AAV-based gene therapy platform.

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