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    Events

  • 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 …

  • World Orphan Drug Congress Asia 2013

    Tuesday, 18 June, 2013

    World Orphan Drug Congress Asia is a strategic 1 day conference and …

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    NanoPass Technologies Supports a CDC-Sponsored Phase 3 Study of Polio Vaccine in Infants as Part of Its Global Health Initiative

    Tuesday, May 14, 2013

    NanoPass Technologies Ltd. ("NanoPass"), a pioneer in intradermal (into-the-skin or ID) vaccine delivery solutions, is collaborating with the US Center of Disease Control and prevention (CDC) in conducting a large phase 3 trial of polio vaccine in infants in South East AsiaThis collaboration is a

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    Onconova Completes Enrollment in ONTIME Pivotal Phase 3 Study of Rigosertib in Patients With High-risk Myelodysplastic Syndromes

    Tuesday, May 14, 2013

    Onconova Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, announced it has reached its enrollment goal of 270 for its pivotal Phase 3 ONTIME trial of rigosertib in patients with myelodysplastic

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    Renaissance in New Drugs for Rare Diseases: Report in World's Largest Scientific Society Magazine

    Tuesday, May 14, 2013

    Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller

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    Isis Pharmaceuticals Earns $3.5 Million From Biogen Idec

    Tuesday, May 14, 2013

    Isis Pharmaceuticals, Inc. announced that is has earned a $3.5 million milestone payment from Biogen Idec associated with the dosing of the first patient in a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMAThe Phase 2 study of ISIS-SMNRx is an open-label, multiple-dose, do

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    Orphans Find a Home

    Monday, May 13, 2013

    One afternoon a week, usually on a Tuesday, a nurse arrives at the Elmwood Park, N.J., home of Jeff and Deena Leider to give their sons, Justin and Jason, their “muscle juice.” On each visit, she carefully inspects a handful of vials, empties them into an intravenous bag, and calibrates a pump

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    30 Years of Orphan Drug Act, Combined With Science and Advocacy, Reflect Dramatic Progress in a Rare Lung Disease, Pulmonary Hypertension Association Says

    Monday, May 13, 2013

    Pulmonary hypertension (PH), a rare and life-threatening lung disease, has gone from rapidly fatal to treatable with nine drugs developed in just the past 20 years, a phenomenal record for a rare condition. Growth in the understanding and treatment of PH reflects the dramatic progress possible Pro

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    United Spinal Secures Victory in Protecting Access to Complex Rehab Technology in Washington State

    Monday, May 13, 2013

    United Spinal Association and other advocates have combined efforts to ensure that people living with disabilities in Washington State have access to properly configured wheelchairs and other mobility equipment or "complex rehab technology"– that is critical to their quality of life and On May 8

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    Lilly Announces Enzastaurin Phase III Study Did Not Meet Primary Endpoint in Diffuse Large B-Cell Lymphoma

    Monday, May 13, 2013

    Eli Lilly and Company announced Phase III clinical trial results from enzastaurin's PRELUDE study, which explored the molecule as a monotherapy in the prevention of relapse in patients with diffuse large B-cell lymphoma (DLBCL). The study failed to show a statistically significant increase compared

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    Alnylam and Genzyme Form Alliance to Develop and Commercialize RNAi Therapeutics in Asia

    Monday, May 13, 2013

    Alnylam Pharmaceuticals, Inc. and Genzyme, a Sanofi company, announced that they have formed an exclusive alliance to develop and commercialize RNAi therapeutics targeting transthyretin (TTR) for the treatment of transthyretin-mediated amyloidosis (ATTR) in Japan and other Asia-Pacific countries.

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    Insmed to Present at the Bank of America Merrill Lynch 2013 Health Care Conference

    Monday, May 13, 2013

    Insmed Incorporated ("Insmed" or the "Company"), a biopharmaceutical company focused on developing and commercializing an inhaled anti-infective to treat patients battling serious lung diseases that are often life-threatening, announced that Will Lewis, President and Chief Executive Officer of A l

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