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Search Results - Orphan Diseases
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  • Isis Wins $2M Milestone Payment from GSK for Antisense Drug Candidate
    indication for which FDA has granted Isis fast-track designation and orphan drug status. ... Isis' pipeline includes 28 drugs designed for a variety of diseases, with emphasis on ...
    7-30-2013
  • Top 20 Corporate Venture Funds
    ... of therapeutic areas of interest including, but not limited to allergy, central nervous system (CNS) and neuropsychiatry, gastrointestinal, hematology, and orphan genetic diseases. ...
    6-14-2013
  • Gene Editing with Multiple Molecular Means
    the approach may offer a giant step forward in the treatment of certain genetic diseases. ... The European Medicines Agency, or EMA, has designated ataluren as an orphan medicinal ...
    6-12-2013
  • Grifols Taking Aradigm Stake in Up To $116M Licensing Deal
    and Lipoquin(tm)) for severe respiratory diseases that include non-cystic fibrosis ... Aradigm holds an orphan drug designation for BE in the U.S. granted by FDA, with no drug ...
    5-21-2013
  • Companion Diagnostics: 52 Pick-Up
    ... may also explore opportunities in infectious diseases, genetics and other conditions." ... which has been designated an orphan drug by FDA and EMA. Financial terms not disclosed. ...
    5-9-2013
  • Pfizer, Alberta Renew Funding for Translational Research
    ... chronic disease issues as they relate to the consequences of diabetes; and orphan and genetic diseases. "Alberta has a vibrant and growing medical research and life sciences ...
    4-25-2013
  • Single-Use Bioreactor Roundup
    ... Other growth areas include personalized medicine, local flex-factory manufacturing sites, and/or biomolecules developed to treat orphan diseases. These applications require smaller ...
    4-15-2013
  • Is This Gene Therapy's Breakout Moment?
    ... fruit such as the orphan diseases accounting for most early indications in gene therapy, and will step up to the plate when there's compelling clinical data relevant to a product. ...
    4-1-2013
  • Exploiting Folate Pathways to Treat Cancer
    ... The EMA granted vintafolide the equivalence of orphan drug status in March 2012, because ... type in inflammatory diseases like rheumatoid arthritis, Crohn's disease, and psoriasis. ...
    4-1-2013
  • Edison Will Receive Up To $525M in Mitochondrial and CNS Disease...
    The initial scope of the transaction includes both pediatric orphan inherited mitochondrial and adult central nervous system diseases. Edison's translational platform is based on ...
    3-28-2013
  • Aastrom Starts Layoffs, Slashes Expenses as Phase III Trial Halted
    protein therapeutics for fibrovascular diseases, and holds more than 20 years of ... our resources on the development of ixmyelocel-T for the DCM orphan indication," he said. ...
    3-27-2013
  • Receptos Licenses AbbVie Antibody for Co-Development
    diseases, said it plans to conduct a Phase II study intended to demonstrate proof-of-concept of the antibody RPC4046 in eosinophilic esophagitis (EoE), an FDA-designated orphan ...
    3-13-2013
  • Is This Gene Therapy's Breakout Moment?
    ... fruit such as the orphan diseases accounting for most early indications in gene therapy and will step up to the plate when there's compelling clinical data relevant to a product. ...
    3-4-2013
  • Genome Editing for R&D and Therapeutics
    ... discovery, because genomics advances tell us that cancer is essentially hundreds of orphan diseases, comprised of multiple subtypes driven by distinct genetic features," says Dr. ...
    3-1-2013
  • Small Firms Look Optimistically to Future
    ... Chaperones Amicus Therapeutics uses pharmaceutical chaperones and oral monotherapy to stabilize patients' own natural enzymes to combat rare and orphan diseases, "especially ...
    3-1-2013
  • More »

    Journal Articles

  • Interleukin-27-Mediated Suppression of Human Th17 Cells Is Associated...
    Hong Liu, Christine Rohowsky-Kochan
    Journal of Interferon & Cytokine Research
    Th17 cells by downregulating retinoid orphan nuclear receptor C expression and that ... may be a promising therapeutic approach in the treatment of Th17-mediated diseases.
  • The Current Status of Drug Discovery Against the Human Kinome
    Richard M. Eglen, Terry Reisine
    ASSAY and Drug Development Technologies
    aimed at treating many devastating diseases, including cancer, autoimmune disorders, ... with the understanding that numerous "orphan" kinases will provide targets for ...
  • Development and Validation of a Cell-Based Assay for the Nuclear...
    Jennifer Wilkinson, Tina Hallis, Spencer Hermanson, Kun Bi
    ASSAY and Drug Development Technologies
    for the Nuclear Receptor Retinoid-Related Orphan Receptor Gamma ASSAY and Drug Development ... and are involved in the pathogenesis of human inflammatory and autoimmune diseases. ...
  • Identification of Signal Transduction Pathways Used by Orphan...
    Janine N. Bresnick, Heather A. Skynner, Kerry L. Chapman, Andrew D. Jack, Elize Zamiara, Paul Negulescu, Kevin Beaumont, Smita Patel, George McAllister
    ASSAY and Drug Development Technologies
    Of the many identified genes encoding GPCRs, >130 are orphan receptors ( i.e. , their ... novel therapeutic targets for pharmaceutical intervention in a variety of diseases. ...

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