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  • Shire marks 30th Anniversary with Innovative Global Program to benefit children with Rare Diseases

    17 February 2016

    To mark the company's founding 30 years ago this year, Shire is announcing a global program intended to benefit children with rare diseases, their families and the rare disease community. Shire is commencing a three-year initiative with global …

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  • China to join the EGA

    10 February 2016

    We are pleased to announce that the Beijing Zhengtao Gaucher Disease Care Center (北京正涛戈谢氏罕见病关爱中心) in China has now joined the EGA as an associate member.

  • The Patient Group Handbook: A Practical Guide for Research and Drug Development

    11 January 2016

    The following book is a must-read for anyone with an interest in orphan drug development and/or re-purposing:…

  • The Psychosocial and Neurocognitive Impacts of Gaucher Disease, Type 1 on Pediatric and Young Adult Patients

    23 November 2015

    Researchers from Palo Alto University are seeking participants between the ages of 5-25 and their caregivers to take part in a study of the psychological and cognitive impacts of Gaucher disease, type 1. The researchers are Andrea Alioto, M.S. and …

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  • Launch of the 'Spotlight on Gaucher Film Contest'

    15 July 2015

      'Spotlight on Gaucher Film Contest'
    has been launched by Shire to encourage those touched by Gaucher disease to share a short video about their experience with the condition. Shire want you to share your own personal story of living with …

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  • UK Gauchers Association Granted a Positive Opinion For Orphan Designation For Gene Therapy For Gaucher Disease

    27 February 2015

    UK Gauchers Association Granted a Positive Opinion For Orphan Designation For Gene Therapy For Gaucher Disease

    27 February 2015
    The UK Gauchers Association is delighted to announce that on 12 February 2015 the Committee for Orphan Medicinal Products …

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  • Watch the Official Rare Disease Day 2015 Video!

    17 February 2015

    Watch and Share the Official Rare Disease Day 2015 Video!

    Bring People Living with a Rare Disease out of the Shadows and into the Spotlight

    Rare disease Day 2015 Video

  • European Commission Grants Marketing Authorization for Cerdelga (eliglustat), Genzyme`s oral therapy for Gaucher Disease Type 1

    23 January 2015

    Paris - January 22, 2015 - Sanofi and its subsidiary Genzyme announced today that the European Commission (EC) has granted marketing authorization for Cerdelga® (eliglustat) capsules, a first line oral therapy for certain adults living with …

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  • European CHMP Adopts Positive Opinion for Genzyme's Cerdelga (eliglustat) Capsules

    23 November 2014

    21.November 2014
    CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme, a Sanofi company, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval …

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