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Search Results - Muscular Dystrophy
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  • Heading, Literally, into the Future
    He maintains that such a procedure would be beneficial-in reality, life saving-for individuals suffering from muscular dystrophy, tetraplegics who are often prone to multi-organ ...
    7-10-2013
  • Large-Scale Small Molecule Screen IDs Muscular Dystrophy Target
    small molecules, scientists have identified a way to 'turn off' the defect behind myotonic dystrophy type 1. Large-Scale Small Molecule Screen IDs Muscular Dystrophy Target
    6-27-2013
  • Gene Editing to Treat Genetic Diseases, Part II
    This second part of a two-part article on treating Duchenne muscular dystrophy discusses TALENs and TAL effectors as possible treatments. Duchenne muscular dystrophy (DMD) and its ...
    6-20-2013
  • NCATS Awards $12.7M for Nine Drug Repurposing Projects
    ... Krieger Institute, and Stanley C. Froehner, Ph.D., of the University of Washington for investigations on an undisclosed Sanofi compound against Duchenne muscular dystrophy (DMD). ...
    6-18-2013
  • Gene Editing with Multiple Molecular Means
    This first part of a two-part article on treating Duchenne muscular dystrophy focuses on a potential new treatment that interferes with nonsense mutations. Using genetic editing, ...
    6-12-2013
  • MDA Gives Valerion's Preclinical MTM Pipeline $1.2M Boost
    The Muscular Dystrophy Association (MDA)'s Venture Philanthropy arm has injected $1.2 million into Valerion Therapeutics' myotubular myopathy (MTM) pipeline, funding the firm to ...
    5-31-2013
  • Top 20 Grant-Giving Disease Foundations
    ... $3.500 million % Awarded as Grants: 89.6% Headquarters: Washington, DC #8. Muscular Dystrophy Association % of Revenues Awarded as Grants: 21.7% 2011 Total Unrestricted ...
    5-21-2013
  • ARMGO Gets $1M for Muscular Dystrophy Drug
    ARMGO Pharma received a $1 million award from the Muscular Dystrophy Association (MDA) for preclinical work in support of an investigational new drug application with the FDA for ...
    4-17-2013
  • Stem Cell Lines
    cancer, Wilm's tumor, and Von Hippel-Lindau syndrome), Huntington's disease, muscular dystrophy (including CMT, FSHD, and Myotonic), and cystic fibrosis as well as some rarer ...
    3-15-2013
  • Parkinson's Collaboration Hopes to Spark New Therapy
    as one million Americans live with the disease, which is more than the combined number of people diagnosed with multiple sclerosis, muscular dystrophy, and Lou Gehrig's disease. ...
    2-27-2013
  • Repligen, Pfizer Make $70M Spinal Muscular Atrophy Deal
    SMA. Repligen's research and clinical efforts, including the current Phase Ib trial, have been partially supported by a $1.4 million grant from the Muscular Dystrophy Association. ...
    1-3-2013
  • The New Economics of Orphan Diseases
    ... At least three compounds are in Phase III trials for rare diseases. An antisense oligonucleotide targets Duchenne muscular dystrophy in collaboration with Prosensa, an ex vivo stem ...
    1-1-2013
  • Isis Gets More Money from Biogen Idec
    I-stage antisense candidate for the treatment of spinal muscular atrophy, ISIS-SMN RX . ... an antisense drug for treating myotonic dystrophy type 1. Biogen Idec and Isis ...
    12-10-2012
  • Shire Puts $22M into Rare Diseases R&D Partnership
    Shire's HGT business is dedicated to the research, development, and commercialization of products for patients with rare diseases, including Duchenne muscular dystrophy, Hunter ...
    10-24-2012
  • Emerging Insights into the Role of the Skeleton
    ... new targets in drug development, especially those designed to treat age-related muscle-wasting disorders and devastating childhood diseases such as Duchenne muscular dystrophy. ...
    8-20-2012
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    Journal Articles

  • Immunity to Adeno-Associated Virus-Mediated Gene Transfer in...
    Zejing Wang, James M. Allen, Stanley R. Riddell, Paul Gregorevic, Rainer Storb, Stephen J. Tapscott, Jeffrey S. Chamberlain, Christian S. Kuhr
    Human Gene Therapy and Part B: Methods
    Immunity to Adeno-Associated Virus-Mediated Gene Transfer in a Random-Bred Canine Model of Duchenne Muscular Dystrophy Human Gene Therapy and Part B: Methods
  • Cell and Gene Therapy in Duchenne Muscular Dystrophy
    Jennifer Elizabeth Morgan
    Human Gene Therapy and Part B: Methods
    Cell and Gene Therapy in Duchenne Muscular Dystrophy Human Gene Therapy and Part B: Methods
  • Clinical Protocol: Phase I Clinical Trial Utilizing Gene Therapy...

    Human Gene Therapy and Part B: Methods
    Clinical Protocol: Phase I Clinical Trial Utilizing Gene Therapy for Limb Girdle Muscular Dystrophy: alpha -, beta -, gamma -, or delta -Sarcoglycan Gene Delivered with ...
  • Flk-1 + Adipose-Derived Mesenchymal Stem Cells Differentiate...
    Yanning Liu, Xi Yan, Zhao Sun, Bin Chen, Qin Han, Jing Li, Robert Chunhua Zhao
    Stem Cells and Development
    Flk-1 + Adipose-Derived Mesenchymal Stem Cells Differentiate into Skeletal Muscle Satellite Cells and Ameliorate Muscular Dystrophy in MDX Mice Stem Cells and Development

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