Recent scientific and medical advances have lead 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 neuromuscular diseases community has undertaken to develop Registries to collate a patient's gene sequence and some key clinical information about their disease.
The NMD 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 improve care of NMD 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 the recruitment process of Australian NMD patients into new clinical trials and by participating in studies for the benefit of the world NMD community and the advancement of medical science.
The Australian Neuromuscular Disorders Registry is guided by the Registry Advisory Committee. This committee includes people from patient organisations as well as experts with relevant clinical, academic or scientific knowledge.The functions of the committee are to guide the development and direction of the Registry and to approve the use of the registry to respond to specific queries from research groups. The Terms of Reference for this committee and the Charter for the Australian NMD Registry are available for your information.
For more information on the Australian Neuromuscular Disorders Registry, please contact the appropriate person from the hospital you attend in the Contact List attached.