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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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    Nanotechnology Could Help Fight Diabetes

    Friday, May 17, 2013

    Injectable nanoparticles developed at MIT may someday eliminate the need for patients with Type 1 diabetes to constantly monitor their blood-sugar levels and inject themselves with insulinThe nanoparticles were designed to sense glucose levels in the body and respond by secreting the appropriate a

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    Connor’s House’ Helps Families Affected by Rare Childhood Disease

    Friday, May 17, 2013

    Some of them are so rare that they affect only one child in a millionAll of them have names the average person couldn't and wouldn't have reason to pronounceThey're all insidious. They are all incredibly rare. And they are all, sadly, almost always fatalWhen families are affected by these rare d

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    RARE DISEASE: Story Co. Sheriff Taken Ill

    Friday, May 17, 2013

    The Story County Sheriff has been diagnosed with a Guillain-Barre syndrome according to a Story County Democrats emailSheriff Paul Fitzgerald was hospitalized in Ames last Wednesday with the disorderIt’s an uncommon illness where a person’s immune system attacks the nerves. Weakness and tingl

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    NPS Pharmaceuticals Recognized by National Organization for Rare Disorders for Gattex® As an Innovative Treatment for Patients With Short Bowel Syndrome

    Friday, May 17, 2013

    NPS Pharmaceuticals, Inc., a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, announced that the company was honored by the National Organization for Rare Disorders (NORD) for the FDA approval of Gattex® (teduglutide [

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    Synageva BioPharma™ Joins the International MPS Network in Support of MPS Awareness Day 2013

    Friday, May 17, 2013

    Synageva BioPharma Corp. (Synageva), a biopharmaceutical company developing therapeutic products for rare diseases, joins the International MPS Network and others around the world in observing MPS Awareness Day 2013. During MPS Awareness day, patient organizations, industry, and other participants

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    Bridport Family Walk to Help Grandson With Rare Genetic Disease

    Friday, May 17, 2013

    THE Hiller family received news just before Christmas that three-year-old Joe had a rare genetic disease that is likely to end his young life by his tenth birthdayIt was undoubtedly the family’s worse Christmas, said grandad Terry from Mead Fields in BridportJoe is one of only 26 children in th

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    United Spinal and the Buoniconti Fund Team Up to Improve Peer Support for People Living With Spinal Cord Injuries and Disorders

    Friday, May 17, 2013

    United Spinal Association and The Buoniconti Fund announced their plans to create a coordinated national network of peer support groups called the "Spinal Network" that will set higher standards in assisting people living with spinal cord injuries and disorders (SCI/DThe goal of the Spinal Network

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    SIMPONI® (Golimumab) Receives FDA Approval for Ulcerative Colitis

    Thursday, May 16, 2013

    NewLink Genetics Corporation, an oncology-focused biopharmaceutical company specializing in immunotherapy, today announced that several clinical presentations on its two proprietary platform technologies have been selected for the upcoming 2013 Annual Meeting of the American Society of Clinical "T

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    Topotarget Announces That an Abstract Containing Positive Final Data From the Registrational BELIEF Study With Belinostat in Peripheral T-cell Lymphoma (PTCL) Has Been Released for Presentation on ASCO.

    Thursday, May 16, 2013

    Final results from the CLN-19 BELIEF study on belinostat in patients with relapsed or refractory (R/R) PTCL show that belinostat is well-tolerated with a favorable safety profile and an objective response rate (ORR) of 26%, cf. the protocol definition of the primary endpoint. Moreover, the data Th

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    Roche’s Obinutuzumab (GA101) Significantly Reduced the Risk of Disease Progression or Death in People With One of the Most Common Forms of Blood Cancer

    Thursday, May 16, 2013

    Roche announced the first results from CLL11, a phase III study of the investigational medicine GA101 which is being conducted in collaboration with the German CLL Study Group (GCLLSG). The CLL11 study compared the combination of either GA101 or MabThera/Rituxan (rituximab) and chlorambucil, a “

    read more>>

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