Recruitment Request: Understanding patients’ and carers’ decision making in Spinal Muscular Atrophy Care

Associate Professor Michelle Farrar and colleagues from Sydney Children’s Hospital (Randwick) are conducting a new research project to lean more about what factors are important when making decisions related to treatment for  spinal muscular atrophy (SMA). They are seeking the views of parents/carers of people with SMA, health care providers and the wider community. Please find attached more […]

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Lifeline for Pompe disease patients

RCH clinicians have worked with the Australian Pompe Association, drug company Genzyme and the Federal government to achieve government-subsidised treatment for late-onset Pompe disease. 12-year-old patient Christian Rivera received his first infusion at the RCH last week. The federal government has listed the only registered treatment for Pompe disease, Myozyme (alglucosidase alfa), on the Life […]

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Development of Allied Health and Nursing Best Practice Guidelines for Duchenne muscular dystrophy

The development of the Duchenne muscular dystrophy clinical practice guideline is currently underway utilising an NHMRC guided process. In keeping with this process, a call for committee members to join the scientific steering group is now open. We would welcome your participation. The guideline development process involves a number of steps. As a snapshot, the […]

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Allied Health and Nursing Alliance (AHNA)

The¬†Allied Health and Nursing Alliance (AHNA) has now been established! We are seeking new members to join our dynamic team to work towards providing one Australasian voice for the Allied Health and Nursing care and research of patients with neuromuscular disorders.¬†Please visit our AHNA webpage for further details: http://www.ann.org.au/allied-health-and-nursing-alliance-ahna/.  

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ANN Congress 2021 – Save the Date

The annual ANN Congress will be on Friday 8th October 2021 at the Larwill Hotel in Melbourne. This will be an in-person congress with numbers strictly limited to 60 attendess. It will be simultaneously streamed by webinar for those unable to attend in person. Draft programme to follow… Registrations will open in August.

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Charcot-Marie-Tooth Clinical Outcome Measure Update

Professor Joshua Burns has launched an updated web-based tool too assist researchers and clinicians to measure the level of disability in Charcot-Marie-Tooth disease (CMT). This new website enables users to rapidly score clinical outcome measures including: the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS), CMT Infant Scale (CMTInfs), CMT Functional Outcome Measure (CMT-FOM), CMT Neuropathy Score (CMTNS) […]

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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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