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) Glycogen storage disease type IIThe scientific name for Pompe disease, based on how glycogen builds up in the body. causes enlargement of

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

Following is background information and a Q & A released by the Pharming/Genzyme LLC on important issues concerning clinical trials

Leiden, the Netherlands/Cambridge, Mass., October 14, 1998—Pharming Group N.V. (Easdaq: PHAR), the Netherlands biopharmaceutical company, and the American biotechnology company

Human Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. 9:1609-1616 (July 20, 1998) Mary Ann Liebert, Inc. Adenovirus-Mediated Transfer of Human Acid Maltase Gene Reduces Glycogen

National Institutes of Health Paul Plotz, M.D., Nina Raben, M.D., Ph.D. Nina Raben, M.D., Ph.D., NIH Research Chemist, visited the

Human Molecular Genetics, January 1998 “Generalized glycogenA stored form of sugar used for energy. storage and cardiomegaly in a knockout mouse modelA laboratory mouse used to study disease and test treatments before human trials. of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart.” by A.