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  • #75566
    Your questions about exon skipping

    Muscular Dystrophy UK wants to take your views and questions regarding the clinical development of exon skipping technology to the European Medicines Agency (EMA) who we will be meeting on 29 April.

    What would you like to ask them? Perhaps you would like to know when EMA is likely to give a drug marketing authorisation? Will there be clinical trials for exon skipping on non-ambulant boys? Or how outcome measures of clinical trials can be widened, to take into account other factors affecting quality of life?

    Please leave a reply below with all your questions and views by 9AM on 23 April.

    The meeting hosted by EMA has been organised by two European Research Networks. It will involve patient representatives, researchers, clinicians and industry representatives. It will be streamed live and you will be able to listen to the discussion. We will let you know any further access details as soon as possible.

    OzgeResearch MDUK
    Posts: 2
    Joined: 20/04/2015
    Re: Your questions about exon skipping

    1. Is exon skipping a cure or another method of managing the disease?
    2. I am a non UK citizen. How can get access to the medicines of exon skipping?

    Posts: 1
    Joined: 21/04/2015
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