The following is a transcript of a telephone interview conducted for the IPA(International Pompe Association) by Kevin O’Donnell of the AGSD-UK (Association for GlycogenA stored form of sugar used for energy. More Storage Disease-United Kingdom. Kevin O’Donnell interviewed Paul Kaplan, General Manager of the...

Excerpt From Press Release—October 5, 2000 Genzyme General and Pharming Group N.V., announced today that results from the first two clinical trialsA research study that tests new treatments or approaches in people. More ever conducted for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More were presented this week at the American Society of Human Genetics (ASHG) meeting in...

Chen focused on the phase I/II trials underway at Duke with 3 IIa infants. Average age at diagnosis is 5 months and survival thereafter is 3 1/2 months on average, with a range of 0-9 months. Very few baby’s survive a year. Recombinant enzymeA protein that helps the body carry out chemical reactions. More from CHO cells (5 mg/kg...

Genzyme Corporation and it’s development partner, Pharming N.V., are committed to providing an effective and practical therapy for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More that can be made available to the greatest number of patients in the shortest possible time. As part of this commitment,...

The Lancet—July 29, 2000 Recombinant human alpha-glucosidase from rabbit milk in Pompe patients Lancet 2000; 356: 397-398 Hannerieke Van den Hout, Arnold J J Reuser, Arnold G Vulto, M Christa B Loonen, Adri Cromme-Dijkhuis, Ans T Van der Ploeg Following is a short...

Genzyme and Pharming to Fund Commercialization Cambridge, Mass., USA, Durham, N.C., USA, and Leiden, the Netherlands, April 19, 2000 - Genzyme General and Synpac (North Carolina) today announced an agreement under which Genzyme will obtain exclusive, worldwide rights...

Pharming’s first Phase II clinical trialA research study that tests new treatments or approaches in people. More for Pompe’s disease finalised, showing survival, skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. More regeneration and overall improvement of heart and lung functions Leiden, the Netherlands, March 15, 2000 The bio-pharmaceutical company Pharming Group N.V....

New York University Medical Center Frank Martiniuk, Ph.D. A collaborative effort to test animal models with enzymeA protein that helps the body carry out chemical reactions. More and gene replacement is underway. Dr. Martiniuk is sending the human gene and acid a-glucosidase to Australia to be tested in March on cattle affected by...

Applied Biological Sciences Systemic correction of the muscle disorder glycogen storage disease type IIThe scientific name for Pompe disease, based on how glycogen builds up in the body. More after hepatic targeting of a modified adenovirus vectorA delivery system, often based on a modified virus, used to carry genetic material into cells. More encoding human acid alpha glucosidase….......... National Academy of Sciences Vol. 96, Issue 16, 8861-8866,...

News Release by Karyn Hede Duke Medical Center News Office August 2, 1999 Using a modified virus to deliver a therapeutic gene, scientists at Duke University Medical Center have shown that, in mice, they can reverse the damage caused by an inherited muscle-wasting...