20th May 2011
Early-bird rate extended!

The early bird rate for the TREAT-NMD conference has been extended to 15th June to allow as many people as possible to take advantage of the lowest rate available.

Moscow myology spring school brings together clinicians from across Russia

Venue: Russian Academy of Civil Service (Российская академия государственной службы), Moscow (www.rags.ru)

18-21 April 2011

Download the program here.

Meeting Report

by Marita Pohlschmidt, UK Muscular Dystrophy Campaign

The value of networking and sharing ideas and experiences between scientists and clinicians was at the heart of the "2nd Spring School of Myology" in Moscow.

From 18-21 April 2011, more than 90 clinicians, scientists and health professionals met at the Russian Academy of Civil Service in Moscow to take part in the second Spring School of Myology. As well as the core of participants from Moscow itself, 11 attendees travelled from St Petersburg and 29 from other parts of the country, including 3 from Vladivostok, 1 from Kharkov, and 1 from Kazan. The purpose of the four-day event was to receive updates on diagnosis, standards of care and therapy development. The course was organised by the patient organisation Nadezhda, the Moscow Region Paediatric Psychoneurological Hospital No.1 and the Moscow Research Institute of Pediatrics and Child Surgery, in collaboration with the ENMC for TREAT-NMD and Andoni Urtizberea for the Paris Summer School.

Following the great success of the first Russian Spring School, which took place last year in St. Petersburg, this course again gave participants with a special interest in the field of neuromuscular disorders the opportunity to exchange their experiences, network and set up collaborations. The inspiring course programme was developed by a Russian/French scientific committee under the leadership of Dr Andoni Urtizberea and consisted of three days intensive training in clinical and genetic aspects of a broad range of conditions, but with a particular focus on the muscular dystrophies, myotubular myopathies and peripheral neuropathies.

A major improvement this year was the introduction of three parallel interactive workshops focusing on diagnosis – the clinical approach, muscle histology and muscle imaging. During these sessions, a short introductory talk was given to small groups of 20 participants who then had the opportunity to scrutinise their expertise through case studies and exercises. The final day was reserved for Russian speakers and the lectures touched on subjects of particular interest to clinicians for improving Russian healthcare services such as rehabilitation and the development of palliative care.

In the spirit of international networking, the school has also facilitated the development of further links between Russian participants and institutions in Western Europe, with the potential for future research collaborations and sharing of expertise. Two of the Moscow participants will also attend the Paris Summer School this coming June.

At last year's event in St Petersburg, most of the participants came from hospitals or research institutions within St. Petersburg and only a few were able to travel from other regions. Several times during the workshop was it pointed out to me that the distances in Russia pose a huge challenge to the establishment of a neuromuscular network. For comparison, Moscow and Paris are 2,484 km apart, while Moscow and Vladivostok have a huge 6,430 km between them, more than double the distance. Nonetheless, some of the participants who had been at the workshop last year fed back this time that particular progress was made during the last 12 months with regards to networking and collaboration. There is now widespread acknowledgement that only if the scientific community and health professionals work together in Russia will they be able to establish the infrastructure to improve the standards of care and speedily bring new technological developments to affected families who are anxious about missing out on the benefit of potential new treatments. Therefore it was fantastic to learn that this year a third of the participants came from outside Moscow, with some having travelled the distance from Vladivostok.

Again this year a Russian patient organisation played a crucial part in the organisation and the co-funding of the event, with additional support being provided by Genzyme. Members from Nadezhda, the muscular dystrophy association active in Moscow, were present throughout the workshop and it was a great opportunity to listen to their stories and to understand where their priorities are. They are currently working together with the clinicians to establish registries for DMD and SMA and boys with DMD are already starting to take part in clinical trials.

In summary, it was a fantastic experience and it was great to learn that the hope expressed last year that the School of Myology would become an integral part of clinical training in Russia was not in vain. On the last day I even heard that planning of a third workshop is already underway. Rotation of the course to one of the regional capitals of Russia will be an additional step to promote the dissemination of myology throughout the country, and Ekaterinburg, Irkutsk and Novosibirsk are some of the candidates under consideration. I do wonder whether there is an ambition to bring the School of Myology to Vladivostok one day.

-- Marita Pohlschmidt

Australasian Neuromuscular Network (ANN) is officially launched
during the Australian Rare Disease Symposium
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The Rare Disease Symposium, held recently in Fremantle, Western Australia, was the ideal setting to officially launch the Australasian Neuromuscular Network (ANN). The ANN is committed to achieving health equity for more than 20,000 individuals living in Australia and New Zealand who are affected by neuromuscular disorders. Given the rare nature of the disorders, and that patients are located all over Australia and throughout New Zealand, a collaborative network such as the ANN is extremely important.

Research into neuromuscular disorders has entered a new era. Advances in sequencing technologies are accelerating gene discovery and our ability to provide an accurate genetic diagnosis. Clinical trials for novel drugs and gene-based therapies are currently underway and hold great promise. The ANN will provide a forum to advance and disseminate information and guide best practice in diagnosis, care and treatment. The best evidence from our clinical and laboratory based research will be quickly translated into best clinical practice. The ANN will promote integrated training programs for clinicians and researchers. Importantly, the ANN will also provide a single voice to advocate for patients and their families. The diagnosis, prevention and treatment of neuromuscular disorders will benefit greatly from a more integrated and united national approach.

The ANN was officially launched in front of an audience of patients, parents, advocacy groups, government and industry representatives, researchers and health professionals. The ANN has also joined the European neuromuscular initiative as a partner of TREAT-NMD – allowing us to gain from and contribute to a global effort. Prof Hanns Lochmüller from TREAT-NMD spoke at the ANN launch and stressed the importance of advances in clinical care and research through international collaboration. For example, the recently established Australian Duchenne muscular dystrophy (DMD) registry connects Australian and New Zealand DMD patients with more than 10,000 patients worldwide across more than 30 countries.

The ANN is structured to address issues relating to clinical care and research effort. Each area has a Steering Committee comprising individuals with expertise in each area, as well as providing representation of all major groups active in the care and study of individuals with neuromuscular disorders across Australia and New Zealand.

ANN Steering Committees:
Clinical Care
Alastair Corbett (Co-Chair, Adults); Kristi Jones (Co-Chair, Paediatrics)
Monique Ryan (Vic); Anita Cairns (Qld); David Mowat (NSW); Rakesh Patel (NZ);Paula Bray (NSW); Michelle Farrar (NSW)
Nigel Laing (Chair)
Nigel Clarke (NSW); Catriona McLean (Vic); Paul Kennedy (Vic); Tom Robertson
(Qld); Peter Taylor (NSW); Michael Buckley (NSW); Mark Davis (WA); Leigh
Waddell (NSW)
Clinical Trials
Andrew Kornberg (Chair)
Monique Ryan (Vic); Kathryn North (NSW); Anita Cairns (Qld); Joshua Burns
(NSW); Phillipa Lamont (WA)
Kathryn North (Chair)
Nigel Laing (WA); Nigel Clarke (NSW); Monique Ryan (Vic); Joshua Burns (NSW); Richard Roxburgh (NZ)
David Jack(Chair)
Deb Robins (Qld); Andrew Kornberg (Vic); Nigel Laing (WA); Kathryn North (NSW); Hilary Rayner (NZ); Julie Cini (Vic); Varlli Beetham (Vic)

International conference on muscle wasting
18th - 23rd September 2011, Ascona, Switzerland
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The International Conference on Muscle Wasting scheduled for 18th - 23rd September will take place in Ascona, Switzerland. Registration opens on June 1st for those who have had their abstracts accepted and others who have expressed an interest in attending.

Places are strictly limited to 120 with organizers expecting demand to exceed supply. If you wish to attend this conference we recommend that you visit the conference website where you will be able to find information about the programme and also up to the minute information about registration.

PPMD Connect conference - early-bird registration ends 1st June
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The conference this year will take place 7th - 10th July in Balitmore MD with the early bird registration fee ending June 1st, 2011.

This annual conference has been part of the Duchenne landscape for almost twenty years now. What started off as a few families that got together to discuss various topics surrounding DMD has now grown into an event that sees over 500 families attending from around the world.

The Parent Project Muscular Dystrophy Annual Connect Conference is the one event in the United States that provides a multi-day gathering where top researchers, clinicians, Duchenne experts, families and friends meet to exchange ideas, meet to learn the latest information and meet to stay connected with a community that refuses to give up hope.

Upcoming meetings
Past newsletters
TREAT-NMD conference 2011
20th May 2011
TREAT-NMD newsletter - 20th May 2011