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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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 trialA research study that tests new treatments or approaches in people. More for Pompe’s disease finalised, showing survival, skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. More 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 enzymeA protein that helps the body carry out chemical reactions. More 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 IIThe scientific name for Pompe disease, based on how glycogen builds up in the body. More after hepatic targeting of a modified adenovirus vectorA delivery system, often based on a modified virus, used to carry genetic material into cells. More 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...
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 and tongue, due to their inability to break down glycogenA stored form of sugar used for energy. More. A team of American scientists...
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 reactions. More replacement therapy now underway in the Netherlands on acid maltase deficiency, Pompe’s disease. Background—Pompe Disease...
Pharming and Genzyme Form Joint Venture to Develop Treatment for Pompe’s Disease
Leiden, the Netherlands/Cambridge, Mass., October 14, 1998—Pharming Group N.V. (Easdaq: PHAR), the Netherlands biopharmaceutical company, and the American biotechnology company Genzyme General (Nasdaq: GENZ), has created a joint venture to develop and commercialize...
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 cells and tissues when it is not broken down properly. More in Skeletal MuscleMuscles that control movement and are commonly affected in Pompe disease. More of Japanese Quail with Acid Maltase Deficiency S. Tsujino 1), N. Kinoshita 1), T. Tashiro 1),...
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 studied the presence of certain disease related...
Netherland’s Scientists Produce Knockout Mouse Model of Pompe disease
Human Molecular Genetics, January 1998 “Generalized glycogenA stored form of sugar used for energy. More storage and cardiomegaly in a knockout mouse modelA laboratory mouse used to study disease and test treatments before human trials. More of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More” by A. J. J. Reuser/A. T. van der Ploeg, et al Enzyme Replacement Therapy (ERT)A treatment that replaces the missing enzyme through IV infusion. More.......... Therapy for Glycogen Storage Disease Type IIThe scientific name for Pompe disease, based on how glycogen builds up in the body. More Acid...
Gene Therapy: Johns Hopkins University
Johns Hopkins University Barry J. Byrne, M.D., Ph.D. Paul D. Kessler, M.D. The following is a summary of the publication in the November 1996 issue of Proceeding of the National Academy of Science, titled “Gene delivery to skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. More results in sustained...
Scientists Make Progress In Gene Therapy For Heart Disease
Johns Hopkin’s University scientists have successfully used a virus to supply a missing gene and its enzymeA protein that helps the body carry out chemical reactions. More product to muscle cells in animals and humans for an extended period. The achievement could have implications in the treatment of an inherited fatal heart...