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Conferences & Workshops International
Workshop A. The role of parent and patient organisations on the global level. Parent and
patient organisations involved in genetic or congenital disorders
such as neuromuscular diseases, are confronted with severe emotional,
psychosocial, physical, and economic implications for which they
have never volunteered nor were educated. Scientific research is unravelling the genetic background of genetic diseases and offers perspectives for early detection, accurate diagnosis, effective therapy and prevention. A substantial reduction of the burden of genetic disease is a current realistic option. Patient organizations can largely and effectively contribute to research by societal (media), political (policy/priority influence), financial (fundraising) interventions and by facilitating scientific groups working in their disease area. Moreover they are experts by personal experience. Each patient is in a way a living database of great value for science if diagnosed properly and well informed. The process
of achieving effective therapy can be accelerated by the support
of parent and patient groups. (f.i. pompe disease ). Parents and patients from all over the world have united themselves in disease bound organisations on a national, continental and global level. They also united on more specific issues such as disability, rarity and genetics. In the neuromuscular area there are over thirty disease specific groups organised on the international level. Some are strong, many are weak. They all focus on the specific interests of their disease. In an increasing number of countries there is a national association for neuromuscular diseases (MDA) which focus on social affairs and on supporting research. On the global level the World Alliance of Neuromuscular Disorder Associations (WANDA) meet quadrennial since 1990 in conjunction with the International Congress on Neuromuscular Diseases. WANDA is represented
in various global organisations to influence policy, priorities
and legislation and increase awareness for the issues considered
of importance by its members such as the promotion of adequate services
for early and accurate diagnosis, appropriate care and understanding
and more attention for the implications of the great many rare disorders. Opinionmaking
takes place on issues such as animal experimentation for pharmaceutical
products, access to medicines, ethical aspects of stemcells, inclusion
of patient representatives in consultation and decision making processes. The role of
parent and patient organisations on the global level is of increasing
importance and well accepted however at the same time, due to lack
of financial and human resources, at this stage limited. Parents
and patient focus in the first place on the specific interest of
the disease to counteract the negative consequences of the disease
which affected them and which often changed their lives. Patient organisations
developed from a situation of helplessness via emancipation towards
management and may grow into a position of being the drivers of
healthcare and promoters of research. B. Developments in Neuromuscular Disease Organisations in the Asia Pacific Region Some four years ago, the idea for forming an Eastern Hemisphere organisation equivalent to the European Neuromuscular Centre (ENMC) was proposed by Alan Emery. As a result, a first Asian and Oceanian Myology Center workshop was held in Tokyo in January 2001, with Hideo Sugita (Japan) as President and Byron Kakulas (Australia) as Vice President. Additional members of the inaugural Executive Board included representatives from the People's Republic of China, India, Indonesia, Japan, The Philippines, Singapore, Taiwan and Thailand. The AOMC aims to promote scientific research into, and to disseminate knowledge about, neuromuscular diseases, by providing relevant educational opportunities especially to young scientists and clinicians so that levels of best scientific and clinical practice can be achieved. Strategies to be employed include the holding of regular scientific/clinical symposia and workshops, to run appropriate teaching courses, to arrange collaborations, to publish appropriate materials and to establish an informative website. Membership of AOMC is open to all scientific, clinical and allied professionals working in the neuromuscular disease field. It is governed by an Executive Board and informed by a broader Advisory Board. A non-profit organisation, its membership is currently free, although it intends future rates to be fixed at $10 for full membership and $5 for associate membership with students remaining free. Several conferences with specific symposia and associated clinico-pathological teaching sessions have now been held since AOMC's establishment. These have been in Chiang Mai, Thailand (mitochondrial myopathies), Beijing, PR China (muscle channelopathies, inclusion body myopathies, distal myopathies) and Singapore (Duchenne muscular dystrophy - new and future therapies, advances in glycogen storage diseases). Many of the countries in the Asia/Pacific region now have strong and growing Neuromuscular Disease societies. Most of these are associated with WANDA and contacts are listed on the "worldmuscleforum" website. They include societies in Australia, China, Guam, India, Japan, Nepal, New Zealand, Papua New Guinea, Pakistan, Singapore, South Korea and Taiwan. In addition to organisations calling themselves "Muscular Dystrophy Associations" or similar names and catering for the broad range of neuromuscular diseases roughly equivalent to the "more than 40 neuromuscular diseases" covered by the MDA of the USA, there are numerous specific disease groups, special interest groups and parent or family organisations. Among the strongest of these are a number of Motor Neurone Disease/ALS societies. Unfortunately, in some instances, individual groups operate in competition rather than in a spirit of cooperation for the good of all. This is nowhere so obvious as in Australia where there have been many unsuccessful attempts to establish a single national Muscular Dystrophy Association, as opposed to the several, long-existing provincial (state) organisations. The result of this has been a failure to gain either telethon support or significant national company sponsorship. It is to be hoped that the long-awaited establishment of an Australian Neuromuscular Disease Foundation that crosses all provincial boundaries will solve some of these problems. International
Workshop on translational research in neuromuscular diseases |
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