President Bush Presents 2007 National Medals of Science and Technology and Innovation Oct 3, 2008
The 2007 National Medal of Technology and Innovation to Roscoe O. Brady, for his discovery of the enzymatic defects and hereditary metabolic disorders such as Gaucher disease, Neimann-Pick disease, Fabry disease and Tay-Sachs disease, devising widely used genetic counseling procedures and the development of highly effective enzyme replacement therapy that provided the foundation of patient treatment; and for stimulating the creation of and fostering the success of many biotechnology companies... (White House News Releases)
VIDEO from Medialink and Shire plc: Rare Diseases Must Be Top of the Health Agenda Feb 29, 2008
Case study: Fabry Disease (also known as Anderson-Fabry Disease) is a lysosomal storage disease with an estimated birth incidence of 1 in 117,000 ... Due to the varied nature of the symptoms and the slow progressive course of Fabry disease, there is an average delay between the onset of symptoms and diagnosis of 12 years ... Life expectancy is generally reduced by 20 years in men and 15 years in women with Fabry disease, compared with the general population. (PR Newswire)
Some question $48m aid package for drug maker Shire's Lexington expansion Feb 14, 2008
Shire's Human Genetic Therapies unit, which develops drugs to address rare medical disorders such as Fabry disease, is based in Cambridge, but needs additional office and laboratory space nearby to accommodate its rapid growth ... Shire's Human Genetic Therapies unit has two key drugs on the market to treat rare diseases: Elaprase for Hunter syndrome and Replagal for Fabry disease. (Boston Globe)
Genzyme 'bullish' on future growth Jan 9, 2008
Fabrazyme, a treatment for Fabry disease, increased 18 percent to $114 million. Genzyme's drugs for rare hereditary disorders treat illnesses caused by a deficiency of certain enzymes needed for critical bodily functions. (Boston Globe)
Beacon Hill leaders promise incentives to Shire Dec 21, 2007
Shire makes several drugs, including Elaprase for Hunter syndrome and Replagal for Fabry disease. As part of the expansion project, the company would move most of its Cambridge operations to Lexington. (Boston Globe)
Amicus Therapeutics to Present at the Lazard Capital Markets Fourth Annual Healthcare Conference Nov 21, 2007
Amicus has two product candidates in Phase II clinical trials, Amigal(TM) for the treatment of Fabry disease and Plicera(TM) for the treatment of Gaucher disease. The Company announced positive data from Phase I clinical trials of AT2220 for the treatment of Pompe disease in October 2007. (PR Newswire)
Profit soars tenfold at Genzyme Corp. Oct 25, 2007
The medicines include Fabrazyme for Fabry disease and Aldurazyme for a genetic condition known as MPS1. Each drug costs an average $200,000 a year per patient, except Myozyme, which costs about $300,000. (Boston Globe)
Shire deal might create 650 jobs Sep 20, 2007
Another of the company's drugs, Replagal, is not yet approved for use by the federal Food and Drug Administration for Fabry disease, a rare genetic disorder causing similar problems. Connie Paige can be reached at. (Boston Globe -- Business)
Genzyme to begin $150m expansion of Allston plant Sep 15, 2007
It produces several drugs to treat rare diseases: Cerezyme for Gaucher disease, Fabrazyme for Fabry disease, and Myozyme for Pompe disease. The plant is also used to package two other products, Aldurazyme, which treats MPS I disease, a genetic disorder; and Thyrogen, a thyroid cancer drug. (Boston Globe)
Combination Therapy Stops Loss Of Kidney Function In Rare Genetic Disease Jul 28, 2007
A combination of two types of blood pressure-lowering drugs--an angiotensin-converting enzyme inhibitor (ACEI) plus an angiotensin-receptor blocker (ARB), added to enzyme replacement therapy (ERT) with agalsidase-beta (Fabrazyme, Genzyme Corporation, Cambridge, MA)--is the first treatment shown to stop progressive loss of kidney function in patients with severe kidney involvement due to the rare genetic disorder Fabry disease, reports a study in the September Journal of the American Society of... (Science Daily)
Genzyme: Geared for Growth Jun 12, 2007
Other marketed ERT products include Fabrazyme, which was approved in the U.S. and Europe for the treatment of Fabry disease, a rare genetic disorder. Fabrazyme sales were $359 million in 2006, up 18% over 2005. (BusinessWeek)
Biotech firms seek space for manufacturing Mar 12, 2007
One of them treats Fabry disease, which causes circulatory problems, pain in the extremities, and kidney breakdown. Another treats children with Hunter syndrome, a rare and often fatal disorder that causes bulging features, growth problems, mental retardation, and serious organ damage. (Boston Globe)
Brandeis and Brigham and Women's Hospital license technology for Gaucher's to Amicus Therapeutics Feb 23, 2007
Amicus is currently conducting Phase 2 clinical trials for its lead compound, Amigal, for Fabry disease, has completed Phase 1 clinical trials of AT2101 for Gaucher disease and is conducting Phase 1 clinical trials of AT2220 for Pompe disease. . (EurekAlert! -- Business News)
Genzyme expects 2007 profit to rise on drug sales Jan 10, 2007
Fourth-quarter sales of Fabrazyme, the company's drug to treat Fabry disease, which causes blood fats to build up in blood vessels, rose 18 percent to $96 million. Sales of the company's biggest drug, Cerezyme, a treatment for Gaucher disease, which causes enlargement of the liver and spleen, rose 13 percent to $262 million. (ABC News -- Wire)
