National Duchenne MD Register
Last modified on 10 Jul 2009 09:31am
Encouraging progress is being made towards establishing a National Duchenne Muscular Dystrophy Register.
The impetus for a voluntary register of all Australian boys with DMD is to provide them and their families with the opportunity to participate in international clinical treatment trials arising from advances in genetic technologies and gene therapy.
For Australian boys with DMD to be considered for inclusion in international clinical trials requires that Australia establish a National DMD Register which holds the necessary clinical information and Dystrophin gene sequence data.
The rapidly increasing knowledge of the genetic basis and molecular pathophysiology of DMD gained over the past decade is now being translated into the development of new therapies which are hoped will improve the treatment of DMD.
The potential benefits of the national DMD register would include enabling coordination of diagnosis, and ensuring that enrolment in clinical trials and therapy and any new intervention strategies are available on an equitable and consistent manner across Australia.
The register would also provide a central resource for sharing information between patients, doctors and researchers.
It is also expected that a national DMD register will further promote a sense of community among affected patients and families and provide ready access to information concerning standards of care, research and available therapies.
The initiative towards the establishment of a national DMD register came from Duchenne Foundation Australia in conjunction with muscular dystrophy associations, support groups and affected families who made representations to state and federal politicians and health ministers.
Following these representations the Federal Parliamentary Secretary for Health and Ageing, Senator Jan McLucas referred the matter of a National Register for Duchenne and Becker Muscular Dystrophy to the Clinical Technical Ethical Principle Committee (CTEPC) of the Australian Health Ministers’ Advisory Council (AHMAC).
The Office of Population Health Genomics (Public Health Division, Department of Health WA) prepared a briefing paper for CTEPC, including background and scoping documents for the development of the national register.
CTEPC members supported the submission and requested the Office of Population Health Genomics convene and lead a Working Group to prepare a report and recommendations paper on establishing a national DMD register.
Now convened, the DMD Working Group includes clinical, diagnostic, research and support group representatives from across Australia.
In May 2009 the DMD Working Group held a meeting of stakeholders attending the Genes for Health conference in Perth.
The Working Group has also been conducting telephone interviews with heads of muscular dystrophy associations and support groups, as well as experts in clinical, diagnostic and research areas of DMD.
The feedback and information received by the Working Group has been very consistent and is being used to inform the development of a preliminary set of recommendations for the National DMD Register. More targeted stakeholder comments will be sought on the document prior to the Working Group finalizing the report and recommendations for CTEPC.
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