TOP NEWS
2024 Acid Maltase Deficiency Association (AMDA)/International Pompe Association (IPA) Conference date has been set!
Dear Pompe Warriors and Patient Community, We are excited to announce the upcoming 2024 Acid Maltase Deficiency Associate - International Pompe Association (AMDA-IPA) Conference, a momentous event dedicated to advancing knowledge, support, and advocacy for individuals...
OTHER NEWS
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 trials for enzyme 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 Therapy 9:1609-1616 (July 20, 1998) Mary Ann Liebert, Inc. Adenovirus-Mediated Transfer of Human Acid Maltase Gene Reduces Glycogen Accumulation in Skeletal Muscle 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 mutations 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 glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease” by A. J. J. Reuser/A. T. van der Ploeg, et al Enzyme Replacement Therapy (ERT).......... Therapy for Glycogen Storage Disease Type II 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 muscle 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 enzyme 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...
Acid Maltase Deficiency Association
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