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    Events

  • 4th ICCCPO Meeting for Parents and …

    24–26 May, 2013

         Dear members and friendswe are pleased to invite you to the 4th …

  • The 2013 EURORDIS Membership Meeting Will …

    30 May – 1 June, 2013

    Every year EURORDIS organises its Membership Meeting in a different …

  • Blood Disorders: European Hematologists …

    13–16 June, 2013

    Hematology is a specialty that covers everything to do with blood: its …

  • Mitral Valve Prolapse (MVP) Dysautonomia …

    14–15 June, 2013

    Al Davies, MD, a leading international expert in physiological …

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    PROLOR BIOTECH RECEIVES NOTICE of ALLOWANCE for NEW U.S. PATENT COVERING LONG-ACTING COAGULATION FACTORS VII and IX

    Tuesday, April 9, 2013

    Nes-Ziona, Israel - PROLOR Biotech, Inc., today announced that it received a notice of allowance from the U.S. Patent and Trademark Office for a new patent application covering the company’s proprietary long-acting CTP-enhanced coagulation factors for the treatment of hemophilia: Factor VIIa-CTP

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    Spectrum Pharmaceuticals Initiates Phase 2 Study of Long-Acting GCSF Drug Candidate, SPI-2012, for the Treatment of Chemotherapy-Induced Neutropenia

    Tuesday, April 9, 2013

    HENDERSON, Nev. - Spectrum Pharmaceuticals, a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, today announced the Company has initiated a Phase 2 clinical program for SPI-2012 (also referred to as "LAPS-GCSF")

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    Pharmacyclics Announces Third Breakthrough Therapy Designation for Ibrutinib From the U.S. Food and Drug Administration

    Tuesday, April 9, 2013

    SUNNYVALE, Calif. - Pharmacyclics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted an additional Breakthrough Therapy Designation for the investigational oral agent ibrutinib as monotherapy for the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic In

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    Orphan Drug Prices Under Siege in Austerity-Minded Europe

    Monday, April 8, 2013

    Treatments for rare diseases are hot properties for drugmakers, who covet the medicines for their exclusive markets, tax breaks and through-the-roof prices. Now that’s changing This year, the Netherlands demanded cuts in the prices of enzyme-replacement therapies including Sanofi (SAN)’s My

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    Positive Preclinical Data for NKTR-214, an Investigational Cancer Immunotherapy Targeting the IL-2 Receptor Complex, Presented at AACR Annual Meeting 2013

    Monday, April 8, 2013

    SAN FRANCISCO - Nektar Therapeutics presented positive preclinical data for NKTR-214, a novel cancer immunotherapy which targets the IL-2 receptor complex, at the 2013 American Association for Cancer Research (AACR) Annual Meeting. NKTR-214 is a new immunocytokine that is being developed as a "We

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    Neuralstem Receives Notice of Allowance of Patent Covering Use of Stem Cells to Treat Amyotrophic Lateral Sclerosis

    Monday, April 8, 2013

    ROCKVILLE, Md. - Neuralstem, Inc., announced that it received a notice of allowance for patent application 12/404,841. This patent covers methods for treating amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) with expanded spinal cord stem cells, including NSI-566. Neuralstem completed a

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    Sarepta Therapeutics Announces Eteplirsen Demonstrates Sustained Benefit on Walking Test Through 74 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

    Monday, April 8, 2013

    CAMBRIDGE, MA - Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today announced updated data from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results at 74 weeks showed a continued stabilization of

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    Understanding and Overcoming an Ultra-Rare Bleeding Disorder

    Friday, April 5, 2013

    Imagine a mother holding her newborn baby in her arms when, suddenly, she notices considerable bleeding from the site of the baby’s umbilical cord. And it simply won’t stop. In moments, her joy and happiness turn into an unimaginable sense of fear and a frantic search for a solutionFor people

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    VG Life Sciences Reports Positive Safety Results From Second Cohort Enrollment in Solid Tumor Clinical Trial

    Friday, April 5, 2013

    SAN MARINO, Calif. - VG Life Sciences, Inc., a biotechnology company focused on leveraging its innovative platform technologies for the discovery and development of drug therapies for cancer, infectious disease, and inflammation, announced today positive results from stage two of its Pre-IND Phase

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    Array BioPharma Announces Updates on ARRY-520 Clinical Data at the 2013 International Myeloma Workshop

    Friday, April 5, 2013

    BOULDER, Colo. - Array BioPharma Inc. announced that results from two ongoing ARRY-520 clinical trials in multiple myeloma (MM) were presented at the 2013 International Myeloma Workshop in Kyoto, Japan. ARRY-520 is a potent, selective KSP inhibitor with a mechanism of action distinct from other Re

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