TOP NEWS
2024 Acid Maltase Deficiency Association (AMDA)/International Pompe Association (IPA) Conference date has been set!
Dear Pompe Warriors and Patient Community, We are excited to announce the upcoming 2024 Acid Maltase Deficiency Associate - International Pompe Association (AMDA-IPA) Conference, a momentous event dedicated to advancing knowledge, support, and advocacy for individuals...
OTHER NEWS
Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk
Article on Transgenic Enzyme Replacement Therapy Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk Van den Hout JM, Reuser AJ, de Klerk JB, Arts WF, Smeitink JA, Van der Ploeg AT. Department of Pediatrics, Sophia Children’s...
Exclusive Interview with Novazyme
This is an approximate transcript (i.e. from notes, not a tape) of an interview between Novazyme and the International Pompe Association (IPA). The participants were, from Novazyme: John Crowley (CEO), Julie Anne Smith (Senior Director, Patient Advocacy), William...
Clinical Trials Genzyme/Pharming
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...
Pharming and Genzyme to Start Study for Pompe’s Disease
Excerpt from Pharming Press Release—March 30, 2001 LEIDEN, the Netherlands, March 30, 2001 - Pharming Group N.V. announced the start of a Phase II-III clinical trial of an enzyme replacement therapy for Pompe’s disease. The trial, under management of the...
Genzyme General and Pharming Report Publication of Study Results for Pompe Disease Therapy
Excerpt From Press Release-March 20, 2001 CAMBRIDGE, MA and LEIDEN,the Netherlands—Genzyme General (Nasdaq: GENZ), a division of Genzyme Corp., and Pharming Group N.V. (AEX: PHAR and EASDAQ: PHAR) announced the publication March 20 in Genetics in Medicine...
Genzyme/Pharming/Synpac—Joint Venture Formed—Technology Purchased
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...
Genzyme General and Pharming Group Report Results From First Two Clinical Trials for Pompe Disease
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...
Excerpt from presentation of Y.T. Chen ,M.D. Ph.D. (Duke University) at ASHG meeting
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...
Statement from Genzyme and Pharming
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,...
Results of First 36 Weeks of Treatment in Infants in Netherlands Published
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...