TOP NEWS
2023 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2023 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
OTHER NEWS
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...
Genzyme General Obtains Rights to Pompe Disease Therapy from Synpac
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
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....
Enzyme Replacement Therapy (ERT), Gene Therapy Diet, Ephedrine, and Exercise Study
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...
Systemic correction of the muscle disorder glycogen storage disease type II
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,...
Duke Scientists Reverse a Rare Form of Muscular Dystrophy in Mice Using Gene Therapy
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...
First IPA Conference Held July 2-4, 1999
The first IPA Conference was held on July 2-4, 1999, in Naarden, the Netherlands. The meeting was attended by over 60 participants from 11 countries which included members of the medical and scientific community, patient organizations and industry. Presentations...
Acid Maltase Deficiency Association
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