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ANN Annual Meeting 6 September 2018

We are looking forward to welcoming you to the annual ANN meeting for another productive and inspiring program. The ANN meeting is taking place on Thursday 6th September 2018 in Sydney. We are delighted to be meeting alongside the Duchenne ACTT Now conference. The Duchenne ACTT now program will certainly be an exciting one with […]

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Recruitment request: SMA patient and family study

The Centre for Community-Driven Research (CCDR*) are partnering with SMA and MD organisations in Australia to conduct a study to understand the experience of those affected by SMA and their needs in relation to future treatment, support, care and information. This study will result in a report that we can all use for future advocacy […]

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Cost-effectiveness of massively parallel sequencing for diagnosis of paediatric muscle diseases

This study demonstrates the cost-effectiveness of investigation using massively parallel sequencing technologies in paediatric muscle disease. The findings emphasise the value of implementing these technologies in clinical practice, with particular application for diagnosis of Mendelian diseases, and provide evidence crucial for government subsidy and equitable access. Please click for┬áthe full paper  

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Early Access Program for Nusinersen

Early Access Program for Nusinersen   There has been a lot of activity in the clinical trial space for the treatment of Spinal Muscular Atrophy (SMA) over the last two years, and Nusinersen, has been identified as the first effective drug to treat SMA Type 1.   Developed in California, Nusinersen (know in the USA […]

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