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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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 […]
Project DMD is a research project focused on exploring the journey to adulthood for young people with Duchenne Muscular Dystrophy (DMD). AHNA researcher Tegan Pinese is coordinating the project to better understand what living as an adult with DMD is like and how young people and their families can best be supported to plan and […]
Getting the support you and your health profesionals need The Foundation is thrilled to release a range of educational toolkits for patients, GP’s and Allied Health Professionals. Our goal is to empower our community when championing for support within the medical world. In September 2015 FSHD Global convened a workshop of 13 leading international and Australian […]
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.