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 Glycogen 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 trials ever conducted for Pompe disease 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 enzyme 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 disease 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 trial for Pompe’s disease finalised, showing survival, skeletal muscle 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 enzyme 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 II after hepatic targeting of a modified adenovirus vector 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...