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  • Novo Nordisk Reports Promising Phase III Results for Hemophilia...
    III trial for N9-GP (glycopegylated recombinant factor IX), for hemophilia B patients. ... a paradigm shift in the treatment of hemophilia B." Novo Nordisk said it expects the ...
    5-17-2013
  • Ipsen Enlists PeptiDream in Search for Therapeutic Peptides
    This deal appears to be potential good news for Ipsen, which recently, along with bankrupt company Inspiration Biopharmaceuticals, sold its rights to hemophilia drugs OBI-1 to ...
    4-10-2013
  • Is This Gene Therapy's Breakout Moment?
    to develop and commercialize potential hemophilia B treatments using gene therapy ... an AAV8 vector in up to 16 adults with Hemophilia B. Pharma is attracted to low-lying ...
    4-1-2013
  • A Good Day for Milestones
    Factor VIII (TheraPEG-FVIII) to treat hemophilia A. Celtic subsequently exercised its ... to 72 hours in a preclinical model of naturally occurring hemophilia A, the firms report. ...
    3-7-2013
  • Is This Gene Therapy's Breakout Moment?
    ... to develop and commercialize potential hemophilia B treatments using gene therapy ... an AAV8 vector in up to 16 adults with hemophilia B. Pharma is attracted to low-lying ...
    3-4-2013
  • Genome Editing for R&D and Therapeutics
    ... a range of monogenic diseases such as hemophilia and lysosomal storage disorders like ... An example is our program that targets hemophilia in which the liver fails to produce ...
    3-1-2013
  • Cangene Inks $300+M Deal for Bankrupt Inspiration's IB1001
    prevention of bleeding in patients with hemophilia B, and two preclinical drug candidates ... factor VIII (rpFVIII) for congenital hemophilia A with inhibitors against human FVIII ...
    2-20-2013
  • Alnylam Losses Widened by Tekmira Settlement
    ... data for several other programs, including ALN-AT3 for hemophilia, ALN-TMP for hemoglobinopathies, and ALN-AAT for liver disease associated with alpha-1 antitrypsin deficiency. ...
    2-8-2013
  • Baxter Reaps Benefits from Inspiration Bankruptcy
    porcine factor VIII (rpFVIII) for congenital hemophilia A with inhibitors against human FVIII and acquired hemophilia A from the bankrupt Inspiration Biopharmaceuticals ...
    1-24-2013
  • Glybera's Story Mirrors that of Gene Therapy
    A number of companies tried to demonstrate that AAV2 could be used successfully in patients. Avigen tried AAV2 for hemophilia, using a muscular or intravenous administration. It ...
    1-1-2013
  • The New Economics of Orphan Diseases
    ... It formed a strategic collaboration with Sangamo BioSciences in February 2012 to develop therapeutics for hemophilia, Huntington's disease, and other monogenic diseases based upon ...
    1-1-2013
  • Firms Project 2013 BLA for Long-Acting Hemophilia A Therapy
    a BLA with FDA for their long-acting hemophilia A therapy during the first half of ... VIII Fc fusion protein (rFVIIIFc) in 165 male hemophilia patients aged 12 years and over. ...
    10-31-2012
  • Inspiration Enters Chapter 11, Seeks Buyer
    promising therapies to people with hemophilia around the globe," John P. Butler, ... families that want to bring important new treatment options to patients with hemophilia. ...
    10-31-2012
  • Biotecnol, PolyTherics Ally on Anticancer Tribody Drug Conjugates
    ... The option exercise follows a feasibility program to develop and evaluate a long-acting PEGylated form of Factor VIII for the treatment of hemophilia A. Biotecnol and PolyTherics ...
    10-23-2012
  • Genzyme to Develop Alnylam's ATTR Drug in Asia
    and partnered programs. Key programs include ALN-TTR, ALN-APC for the treatment of hemophilia, ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment ...
    10-22-2012
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    Journal Articles

  • Orchiopexy-Laparoscopy or Traditional Surgical Technique in Patients...
    Pastor Escarcega-Fujigaki, Guillermo Hernandez-Peredo Rezk, Edgar Huerta-Murrieta, Nancy Lezama-Ramirez, Santiago Hernandez-Gomez, Gerardo Kuri-Cortes, Abimael Bañuelos-Montano
    Journal of Laparoendoscopic& Advanced Surgical Techniquesand Part B, Videoscopy
    There were no accidents with any of the techniques, and 1 patient with the open technique had an important hematoma; hemophilia was later diagnosed in the patient. The esthetical ...
  • Factor VIII Can Be Synthesized in Hemophilia A Mice Liver by...
    Neelam Yadav, Sumod Kanjirakkuzhiyil, Mallika Ramakrishnan, Taposh K. Das, Asok Mukhopadhyay, Sumod K, Mallika R
    Stem Cells and Development
    Factor VIII Can Be Synthesized in Hemophilia A Mice Liver by Bone Marrow Progenitor Cell-Derived Hepatocytes and Sinusoidal Endothelial Cells Stem Cells and Development Hemophilia ...
  • Cytokine-Conditioned Dendritic Cells Induce Humoral Tolerance...
    Gautam Sule, Masataka Suzuki, Kilian Guse, Racel Cela, John R. Rodgers, Brendan Lee
    Human Gene Therapy and Part B: Methods
    This is best exemplified by inhibitor formation in the protein therapy for hemophilia A. An approach to overcoming this is induction of immunological tolerance to the therapeutic ...
  • Long-Term Expression of Human Coagulation Factor VIII in a Tolerant...
    Christopher L. Chavez, Annahita Keravala, Jacqueline N. Chu, Alfonso P. Farruggio, Vanessa E. Cuéllar, Jan Voorberg, Michele P. Calos
    Human Gene Therapy and Part B: Methods
    Abstract We generated a mouse model for hemophilia A that combines a homozygous knockout ... method to achieve long-term FVIII gene therapy in a relevant mouse model of hemophilia A.

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