Recent scientific and medical advances have led to substantial changes in the treatment of many neuromuscular diseases (NMD). New therapeutic strategies are being developed and, for some of these treatments, plans for large studies involving patients from more than one country are already in place.
The Australian National Duchenne Muscular Dystrophy (DMD) Registry has been developed to collate a patient's DMD gene sequence and clinical information about their disease. The Registry provides an important enabling tool for clinicians and clinical trial sponsors to quickly identify patients suitable for each study, particularly those therapeutic strategies that target specific genetic defects. Potentially eligible patients will be informed about new trials and studies through the Registry's clinical network.
To provide Australian Duchenne Muscular Dystrophy families with an opportunity to improve the outcomes of individuals affected by DMD.
To improve care of DMD patients through the coordination of diagnosis and therapy and by ensuring new intervention strategies are available on an equitable and consistent manner across Australia; and
To improve opportunities for international collaboration by facilitating and accelerating recruitment process of Australian DMD patients into new clinical trials and for participating in studies for the benefit of the world DMD community and the advancement of medical science.
The Australian National DMD Registry adheres to the National Health and Medical Research Council's National Statement, forms part of an international effort led by TREAT-NMD, which brings together patients and specialists working on treatments for neuromuscular (NMD) disorders, and has received approval reference #2010/23 from the Department of Health WA Human Research Ethics Committee.