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...

Duke University Starts Clinical Trials for Pompe’s Disease

The following status statement was released by Synpac (North Carolina), Inc., on June 30, 1999 The following status statement was released by Synpac (North Carolina), Inc., on June 30, 1999 Synpac Pharmaceuticals Limited Cambois, Bedlington, Northumberland NE22 7DB...

Modified Adenovirus for GSD-II

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. More causes enlargement of certain muscles, particularly the heart...

General Information On Clinical Trials

Orphan Drug Designation A drug becomes an “orphan” when it receives orphan designation from the Office of Orphan Products Development, FDA. An orphan drug is a drug intended to treat diseases or conditions affecting fewer than 200,000 people per year in the United...

Phase II Pilot Trials Begin (January 1999) in the Netherlands

Following is background information and a Q & A released by the Pharming/Genzyme LLC on important issues concerning clinical trialsA research study that tests new treatments or approaches in people. More for enzymeA protein that helps the body carry out chemical...

Human Gene Therapy 9:1609-1616

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

Gene Therapy: National Institutes of Health

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 Netherlands to collaborate with Dr. Arnold Reuser on genetic mutationsA change in a gene that can affect how it works. More in AMD. They...