Submission + - Genetherapy effective for patients with LPL (amtbv.com)
Tjeerd writes: "Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, today announced that its lead product, AMT 011, has received Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of Lipoprotein Lipase (LPL) Deficiency.
In a study of eight subjects with Complete LPL deficiency presented on May 30, 2007 at the 10th Annual Meeting of the American Society of Gene Therapy, AMT's lead product was shown to be safe and efficacious."
