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2009 OCT 12 ... (1-deoxynojirimycin HCl), its investigational drug in development for the treatment of Pompe Disease. The primary objective of this study is to evaluate the pharmacokinetics of AT2220 in ...
2009 MAR 9 ... trial of its investigational drug AT2220 (1-deoxynojirimycin HCI) for the treatment of Pompe Disease and that it has received verbal notice from the U.S. Food and Drug Administration (FDA) ...
2009 AUG 3 ... According to a study from Netherlands, "Enzyme analysis for Pompe disease in leukocytes has been greatly improved by the introduction of acarbose, a powerful ...
2009 JUN 8 ... "Lysosomes filled with glycogen are a major pathologic feature of Pompe disease, a fatal myopathy and cardiomyopathy caused by a deficiency of the glycogen-degrading ...
2009 MAY 18 ... review.
In addition, the FDA has agreed that clinical data from Genzyme’s Pompe patient registry can now fulfill the requirements for a verification study. Genzyme is ... a phase 2 study of GENZ-112638, an oral therapy in development for patients with Gaucher disease type 1. After one year, the data showed that the compound improved or stabilized the ...
2009 MAR 16 ... regarding its application to market Lumizyme™ (alglucosidase alfa) for the treatment of Pompe disease. In its letter, the agency outlines the remaining items that need to be addressed before ...
2009 MAR 2 ... but it is not obliged to do so. About Myozyme Myozyme is the only approved treatment for Pompe disease, a progressively debilitating disease that manifests as a broad spectrum of clinical ...
2009 JUN 8 ... The submission included clinical data requested by the FDA from Genzyme’s Pompe Registry. The FDA has agreed that these data can fulfill the requirements for a ... The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing. ...
2009 JUN 8 ... of breathing problems in children with a severe form of muscular dystrophy known as Pompe disease.
Researchers at the Powell Gene Therapy Center at the University of ...
2009 SEP 7 ... to enzyme replacement therapy with recombinant human acid alpha glucosidase in infantile Pompe disease. Eighteen patients,:56 months old, were enrolled in a clinical trial of enzyme ...
2009 AUG 3 ... of enzyme replacement and enzyme enhancement by pharmacological chaperones in Pompe disease (PD), a metabolic myopathy caused by the deficiency of the lysosomal acid ...
2009 MAY 13 ... the safety and efficacy of alglucosidase alfa in infants and children with advanced Pompe disease. Open-label, multicenter study of IV alglucosidase alfa treatment in 21 infants 3-43 ...
2009 AUG 10 ... According to a study from the United States, "Pompe disease is a severe form of muscular dystrophy due to glycogen accumulation in all tissues, ...
2009 AUG 31 ... assaying the enzymatic activities on dried blood spots from 10 patients affected by ''Pompe'', 6 by ''Gaucher'', 12 by ''Fabry'', 3 by ''Niemann-Pick'' A/B, and 2 by ... normal range. In heterozygous carriers (18 for Fabry, 10 for Pompe, and 4 for Gaucher disease) the activities were slightly lower than in control subjects," wrote G. Lamarca and ...
2009 AUG 5 ... in alpha-glucosidase cause accumulation of glycogen in lysosomes, resulting in Pompe disease, a lysosomal storage disorder. Small molecule chaperones that bind to enzyme proteins ...
Subject: Plastic Reconstructive and Aesthetic Surgery
2009 FEB 23 ... be called Lumizyme™ (alglucosidase alfa), giving the company the ability to promote the Pompe disease therapy produced at this scale in the U.S. market. Mozobil™ (plerixafor injection), ...
2009 FEB 23 ... to recent research from Paris, France, "Glycogen storage disease type II (GSDII) or Pompe disease is an inherited disease of glycogen metabolism caused by a lack of functional lysosomal ...
2009 AUG 4 ... intervention with a targeted enzyme as important considerations in the management of Pompe disease." Zhu and colleagues published the results of their research in Molecular ...
2009 NOV 1 ... for its first drug candidate, ZC-701, an enzyme replacement therapy for the treatment of Pompe disease. Jonathan LeBowitz, Chief Scientific Officer of ZyStor Therapeutics, Inc., ...
