Duchenne Muscular Dystrophy

News Duchenne Muscular Dystrophy

• Pioneering research into Duchenne muscular dystrophy secures commercial funding

  Royal Holloway is giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign. Families affected by the condition are also visiting laboratories on campus to find out from the scientists about new drug developments and see the work being carried out. Af more

• Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy

  Prosensa has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted orphan drug designation for two additional DMD compounds in its pipeline.Prosensa identified suitable oligonucleotide candidates for PRO052 and PRO055, designe more

• Big Pharma and Biotech Increasingly Tackling Rare and Niche Diseases

  Rare and niche diseases are being increasingly spotlighted by pharmaceutical and biotechnology corporations in the hope of developing initial treatments or improving upon existing therapies for these conditions, according to a new report released by GlobalData.The new report, "Rare and Niche Disease more

Publications Duchenne Muscular Dystrophy

• N-Glycolylneuraminic acid deficiency worsens cardiac and skeletal muscle pathophysiology in {alpha}-sarcoglycan-deficient mice

  Roughly 3 million years ago, an inactivating deletion occurred in CMAH, the human gene encoding CMP-Neu5Ac (cytidine-5'-monophospho-N-acetylneuraminic acid) hydroxylase (Chou HH, Takematsu H, Diaz S, Iber J, Nickerson E, Wright KL, Muchmore EA, Nelson DL, Warren ST, Varki A. 1998. A mutation in hum more

• Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy

  Duchenne muscular dystrophy (DMD) is characterized by severe degeneration and necrosis of both skeletal and cardiac muscle. While many experimental therapies have shown great promise in treating skeletal muscle disease, an effective therapy for Duchenne cardiomyopathy remains a challenge in large a more

• Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

  Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Molecular Therapy advance online publication, June 4 2013. doi:10.1038/mt.2013.111 Authors: David G Ousterout, Pablo Perez-Pinera, Pratiksha I Thakore more

Market studies Duchenne Muscular Dystrophy

• Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012

  Introduction GlobalData’s new report, "Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012" takes a look at rare and niche diseases, which are an area of intense interest in the global biopharmaceutical industry today. The passage of the Orphan Drug Act in the United States in 19 more

• Duchenne Muscular Dystrophy – Pipeline Review, H2 2012

  Introduction Global Markets Direct’s, 'Duchenne Muscular Dystrophy - Pipeline Review, H2 2012', provides an overview of the Duchenne Muscular Dystrophy therapeutic pipeline. This report provides information on the therapeutic development for Duchenne Muscular Dystrophy, complete with latest update more

• Duchenne Muscular Dystrophy Global Clinical Trials Review, H2, 2012

  Introduction GlobalData's clinical trial report, “Duchenne Muscular Dystrophy Global Clinical Trials Review, H2, 2012" provides data on the Duchenne Muscular Dystrophy clinical trial scenario. This report provides elemental information and data relating to the clinical trials on Duchenne Muscular more

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