Our Approach

Duchenne Australia Focus on 4 Key Pillars

Care and Support

• Achieving evidence based best standards of care for all Australians living with Duchenne or dystrophinopathies.

• Provide information and support for the ‘Lifespan’ from diagnosis, through transition and beyond, developing a focus on ‘Care Across the Lifespan.’ This will be achieved by provide the community with evidence based care standards pertaining to Duchenne and associated dystrophinopathies that can inform the clinical community and empower all of those living with a dystrophinopathie and their families / carers to ensure they have access to the Australian perspective of the international care considerations from TREAT-NMD.

• Focus on mental health of those living with Duchenne their families, carers and importantly siblings – through new diagnosis and for the lifespan, helping develop resilience and supportive strategies.

• To provide the Australian community with regular peer support and networking opportunities through webinars, zoom and face to face events.

Awareness and Education

• Develop community awareness strategies, social media campaigns and fundraising programs.

• Sharing evidence-based resources across the community.

• Work alongside the Government to ensure bipartisan support for our community and the wider rare disease community to guide the development of policies that focus on bridging the gaps in standards of care, access to emerging therapies, quality of life and support services and medical technologies.

• Zoom webinar calendar of educational and networking events.

• Strengthening and developing national and international collaborations with likeminded organisations with the same goal for our community and wider rare disease community.

• Work to increase awareness and advocacy for early diagnosis and a national newborn screening program for Duchenne.

Research and Data

• Focus on the collection and utilisation of robust data through the Australian Neuromuscular Disease Registry (ANMDR) and wider rare disease community through national and international collaborations, pertaining to patient experience data, HTA process, and the Australian perspective.

• Work with government and strategic collaborations to gather patient experience evidence data to inform changes in practice and add to the knowledge.

• Seek to develop new research opportunities in the areas of new therapies or advancing scientific knowledge and research in to therapeutic advancements, quality of life enhancing initiatives / standards of care and newborn screening for Duchenne.

• Working with research institutions nationally and internationally to maximise research opportunities and advocate for increased research investments in Duchenne, dystrophinopathies and rare diseases.
  

Advocacy and Collaboration

• Develop strategic alignments with national and international likeminded organisations to achieve our common goals for the community.

• Advocate to State and Federal Government:

- By raising awareness of Duchenne and associated dystrophinopathies, and the lived

experience of the Australian community.

- Advocating for improved access to clinical trials,

- For a commitment to increase the funding for research into dystrophiopathies,

- For improved / timely processes for the community to access emerging therapies through the

HTA process and associated regulatory processes in Australia.

• Working collaboratively in the National advocacy for all those living with Rare Diseases.

• Advocating and working alongside industry to ensure all Australians have the latest information pertaining to clinical trials and emerging therapies, and information pertaining to the Australian perspective of Duchenne and associated dystrophinopathies.
  

• To advocate for equitable access to best practice standards of care for all living with a dystrophinopathies across Australia.

• To advocate for improved processes and equitable access to the National Disability Insurance Scheme (NDIS) for all those living with dystrophinopathies and their families / carers.