Duchenne Australia are committed to advocating, supporting and driving world class research to achieve our mission and ultimately find effective treatments and a cure for all.
Research is one of the key focus areas of our organisation. We aim to:
Focus on the collection and utilisation of robust data through the Australian Neuromuscular Disease Registry and wider rare disease community through national and international collaborations, pertaining to patient experience data, HTA process, and the Australian perspective.
Work with government, industry and strategic collaborations to gather patient experience evidence data to inform changes in practice and clinical care, to inform protocol development and expediate bench to bedside research, whilst adding to the knowledge base nationally and internationally.
Seek opportunities to develop new research opportunities in the areas of quality of life enhancing initiatives / standards of care and newborn screening for Duchenne and associated dystrophinopathies.
If you are a person or family living with Duchenne or an associated dystrophinopathie (this means if you or your child / children have Duchenne or Becker or are a carrier of these conditions), then you are eligible to enrol in the Australian Neuromuscular Disease Registry (ANMDR) based at the Murdoch Children's Research Institute (MCRI). Your data is essential to build a comprehensive database, to inform decision makers, clinicians, researchers, and industry nationally and internationally to work in collaboration to discover new therapies, inform the decision makers on access to new therapies and the care, quality of life needs of our community, and advance scientific knowledge.
One of our key aims is to make research clear and understandable. We've prepared the glossary below, which covers many of the medical terms used by medical researchers and professionals.
Come chat to us, we are always happy to take enquiries.