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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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 the full paper
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 […]
There has been a lot of activity in the clinical trial space for spinal muscular atrophy in the last 2 years. Nusinersen is an antisense oligonucleotide, or a short synthetic stretch of nucleic acid, that is designed to specifically bind SMN2 transcripts and “correct” SMN2 gene expression. Two hospitals in Australia- the Royal Children’s Hospital […]
Our goal is to ensure excellence in diagnostic methods and clinical management, and equal access to clinical trials and new therapies, for all individuals in Australia and New Zealand affected by neuromuscular disorders.
We can achieve our vision by establishing a cohesive, integrated neuromuscular network which enables people to work together across Australia and New Zealand, for the well-being of patients.