On Thursday February 25th 2010, a group of 33 clinicians, pathologists, geneticists, researchers and patient advocates from around Australia and New Zealand met to discuss how to network more effectively for diagnosis, research, therapy and advocacy of patients with neuromuscular disorders.
- The neuromuscular community within Australia has a very positive history of research collaboration involving clinical and pathological studies of large patient cohorts, genotype-phenotype correlation, gene discovery, the coordination of diagnostic workups between diagnostic and research labs and, more recently, the establishment of disease registers and clinical trials. The participants in the meeting had a history of interaction and collaboration in one or more of these areas.
- Over the past few years there has been increasing discussion about the need for an integrated diagnostic networkfor these rare disorders – so that diagnosis is incorporated into diagnostic lab workup appropriately and in a cost-effective manner. The diagnosis of many of these disorders still relies on the research interests of individual labs and is not adequately funded.
- Over the past few years, Australian patients have participated in international trials through the establishment of Clinical Trials Centres in Melbourne and Sydney as part of the Cooperative International Neuromuscular Research Group (CINRG) and in partnership with PTC Therapeutics
- In 2009, at the New Zealand MDA Conference attended by Professor Katie Bushby (Newcastle-upon-Tyne, UK and TREAT-NMD), there were initial discussions about the integration of Australian and New Zealand efforts as a single entity into the TREAT-NMD network.
- Over the past 12 months, there has been a collaborative effort to establish an Australian national register for patients with Duchenne Muscular Dystrophy to facilitate involvement in clinical trials.
The Towards a Brighter Future Conference provided an opportunity to have everyone in the same place at the same time to have a face-to-face discussion of the “Coalition of the Willing” (Nigel Laing 2010). When Katie Bushby agreed to attend the conference, it provided a wonderful opportunity to consider the establishment of a formal network affiliated with TREAT-NMD. During the introductions at the Workshop – everyone was asked to talk about “their passions” in the fields of diagnostics, research, clinical care and advocacy. The following things were immediately evident:
- We have many areas of complementary expertise.
- Collaboration will increase our productivity, ability to “make things happen”, ability to provide advocacy and ability to attract funding from Government.
- There is room and increased opportunity within a network for everyone to pursue their major interests and passions and to share expertise. Any individual at any site would be enabled to “plug in” to the network to enhance their own research and studies
- There is a huge amount to do
- We could easily expand into many areas – but the first priority is to get us working together effectively as a group (baby steps)
By the end of the first session we had reached consensus about the need to establish a network