A new weapon to fight cancer - tobacco? Aug 22, 2008
Plant-based biotechnology already has been used to produce drugs for diseases such as cystic fibrosis and Gaucher's disease, but some of these personalized drugs can be pricey for consumers. Avastin, a cancer drug made by South San Francisco-based Genentech, can cost up to $8,800 per month, and Genzyme's Cerezyme, which is used for Gaucher disease, costs about $200,000 a year. (AZCentral -- Business)
AIIMS: Babies die during clinical trials Aug 18, 2008
They were zinc tablets for treating zinc deficiency and serving as a nutritional supplement, olmesartan and valsartan for treating blood pressure-related problems, rituximab for treating chronic focal encephalitis and gene-activated human glucocerebrosidase for treating Gaucher's disease, which affects the liver. AIIMS said it had taken clearance for the trials from its own ethics committee, the health ministry steering committee (HMSC) on ethics and the national ethics committees of ICMR and... (India Times, India)
Skin Cells Produce Library of Diseased Stem Cells Aug 12, 2008
Other cell types they have made to date include samples from people with Huntington's disease, a form of combined immunodeficiency commonly known as "bubble boy's disease," Lesch-Nyhan syndrome, Gaucher's disease, and two forms of muscular dystrophy. Reuters Health. (MEDLINEplus)
Twenty Disease-specific Stem Cell Lines Created Aug 9, 2008
The cell lines the researchers produced carry the genes or genetic components for 10 different diseases, including Parkinson's Disease, Type I diabetes, Huntington's Disease, Down Syndrome, a form of combined immunodeficiency ("Bubble Boy's Disease"), Lesch-Nyhan syndrome, Gaucher's Disease, and two forms of Muscular Dystrophy, among others. "We wanted to produce a large number of disease models for ourselves, our collaborators, and the stem cell research community to accelerate research," Daley... (Science Daily)
Catalogue of diseased stem cells unveiled Aug 8, 2008
Other cell types they have made to date include samples from people with Huntington's disease, a form of combined immunodeficiency commonly known as "bubble boy's disease," Lesch-Nyhan syndrome, Gaucher's disease, and two forms of muscular dystrophy. Recommend this article. (Globe and Mail)
Axway Inc. will track drugs for Genzyme May 12, 2008
Its biggest sellers are Cerezyme for Gaucher's disease, a metabolic disorder, and Renagel for kidney disease. Genzyme had previously used the gateway portion of Axway's offerings. (AZCentral -- Business)
Cost-control plan: generics Mar 3, 2008
Cambridge-based has developed a portfolio of life-saving medications to treat a number of rare illnesses, such as Pompe disease and Gaucher's disease. But the drugs come at a steep price: $200,000 to $300,000 a year. (Boston Globe)
Scientific and Legal Viability of Follow-on Protein Drugs Feb 21, 2008
1 Though small-molecule drugs are more common than recombinant-protein drugs only 1 of the top 200 prescribed drugs of 2006 (on the basis of prescription volume) was a recombinant protein protein-based therapeutics have been used to treat diabetes and anemia, as well as relatively rarer conditions, such as rheumatoid arthritis, Gaucher's disease, and multiple sclerosis. 2,3. (New England Journal of Medicine)
Chemical Chaperone Could Open Door To Treatment Of Neurological Disorder Feb 10, 2008
(May 29, 2006) Prospects for developing a less expansive and more convenient treatment for Gaucher's Disease have brightened with the discovery that certain small molecules can partially correct the underlying. . (Science Daily)
Genzyme stock up on takeover rumors after Icahn buy Nov 16, 2007
The company, with a market value of close to $19 billion, generates billions of dollars in sales from treatments for rare disorders, including Gaucher's disease and Pompe disease. Todd Wallack can be reached at. (Boston Globe)
Icahn reports interest in Genzyme Nov 15, 2007
The company, led by industry veteran Henri Termeer, is known for producing powerful treatments for rare disorders, such as Gaucher's disease and Pompe disease. Icahn hasn't publicly stated whether he plans to increase his stake in the company or push for any changes. (Boston Globe)
Profit soars tenfold at Genzyme Corp. Oct 25, 2007
2 million on the strength of its biggest product, Cerezyme, a treatment for Gaucher's disease, and its newest drug for hereditary disorders, Myozyme, for Pompe disease. Excluding costs, results beat analysts' estimates by 4 cents. (Boston Globe)
Gene Abnormality Tied To Getting Parkinson's Disease At A Younger Age Sep 19, 2007
Mutations in the GBA gene cause Gaucher's disease, which is a rare disorder that prevents organs, such as the spleen and brain, from working properly due to the build-up of a fatty substance called glucocerebroside. "Our results confirm that GBA mutations are risk factors for Parkinson's disease and may lead to getting the disease at a younger age," said study author Lorraine N. Clark, PhD, and coauthor Karen Marder, MD, MPH, with Columbia University's Taub Institute for Research on Alzheimer's... (Science Daily)
Raytheon profit up on missile sales Apr 26, 2007
2 million on the strength of therapies for rare genetic disorders such as Cerezyme, a treatment for Gaucher's disease, and its newest product, Myozyme, which treats Pompe disease. Medicines for rare diseases account for almost half of the company's revenue. (Boston Globe)
Blood-brain barrier bypassed Apr 25, 2007
In humans, a deficiency of this enzyme causes Gaucher's disease, a genetic disorder that is often neurodegenerative and fatal ... The barrier makes neural disorders such as Alzheimer's and Gaucher's diseases very hard to treat, since existing drug-delivery techniques -- such as brain injections, implants, and nanoparticles -- can bring only small quantities of protein into the brain. (The Scientist)
Lack Of GBA2: A Contraceptive For Male Mice Nov 3, 2006
Individuals lacking the GBA2 related protein GBA1 suffer from Gaucher's disease (a disease that causes various symptoms, including enlarged internal organs and neurologic defects) and one treatment for this disease has been shown to decrease fertility in mice. This study, identifying a crucial role in male fertility for GBA2, might help explain the effect on fertility of this treatment for Gaucher's disease and, as Martin Matzuk and colleagues explain in an accompanying commentary, might help... (Science Daily)
Genzyme stock climbs on strong earnings Jul 13, 2006
In the past quarter, Genzyme sold $254 million worth of its flagship drug, Cerezyme, an injectable treatment for an uncommon genetic disorder called Gaucher's disease. That represented an 8 percent increase over second-quarter sales of Cerezyme last year. (Boston Globe)
Groups and Genes Jun 21, 2006
People with the genes for torsion dystonia, non-classical congenital adrenal hyperplasia, and Gaucher's disease tend to have higher average IQs, or tend to be concentrated in professions such as physics and engineering. But the numbers are small. (Ocnus.net)
Toward a less expensive, more convenient treatment of Gaucher's disease May 25, 2006
Prospects for eventual development of a less costly and more convenient treatment for Gaucher's disease have brightened with new research findings reported in the May issue of ACS Chemical Biology ... Individuals with Gaucher's disease, which can be fatal, produce a defective form of GC, a critical enzyme that breaks down a fatty substance called glucosylceramide ... In the new report, scientists have confirmed experiments they reported initially in 2002 that "chemical chaperones" can partially... (EurekAlert!)
