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

The first IPA Conference was held on July 2-4, 1999, in Naarden, the Netherlands. The meeting was attended by over

The following status statement was released by Synpac (North Carolina), Inc., on June 30, 1999 The following status statement was

The following article by Laura Spinney appears in BioMednews Reports (6- 25-99,#57) GlycogenA stored form of sugar used for energy. storage disease type II causes enlargement of

Orphan Drug Designation A drug becomes an “orphan” when it receives orphan designation from the Office of Orphan Products Development,