The following is a transcript of a telephone interview conducted for the IPA(International Pompe Association) by Kevin O’Donnell of the
October 6, 2000
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
Chen focused on the phase I/II trials underway at Duke with 3 IIa infants. Average age at diagnosis is 5
October 1, 2000
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
The Lancet—July 29, 2000 Recombinant human alpha-glucosidase from rabbit milk in Pompe patients Lancet 2000; 356: 397-398 Hannerieke Van den
Genzyme and Pharming to Fund Commercialization Cambridge, Mass., USA, Durham, N.C., USA, and Leiden, the Netherlands, April 19, 2000 –
Pharming’s first Phase II clinical trialA research study that tests new treatments or approaches in people. for Pompe’s disease finalised, showing survival, skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. regeneration and overall improvement of heart
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. and gene replacement
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. after hepatic targeting of a modified
News Release by Karyn Hede Duke Medical Center News Office August 2, 1999 Using a modified virus to deliver a
