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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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UPDATE ON SMA RESEARCH AND TREATMENT- AUSTRALIA

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 […]

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Project DMD

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 […]

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Update: evidence based guideline summary for FSHD

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 […]

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