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31st May 2013
TREAT-NMD Alliance meeting - save the date!

We are delighted to announce a dedicated meeting of the TREAT-NMD Alliance to be held in Newcastle, UK from 30 October - 1 November 2013.

This meeting is a fantastic opportunity for patients, academics, clinicians, patient registry curators and industry representatives, indeed all TREAT-NMD stakeholders, to get together to network and exchange ideas and learn about new research.

Highlights of the meeting will include a look at the scientific results and achievements that TREAT-NMD related research has contributed to, along with a session on upcoming themes of collaborative research. The meeting also includes a session to discuss the role of patients in research and related ethical questions.

To maximize the benefit of so many TREAT-NMD stakeholders being together at the same time a number of peripheral meetings have also been organized. These include a TGDOC meeting (registry curators welcome), TACT Core Group meeting and a meeting of the TREAT-NMD Alliance Executive Committee.

Further details of the programme, venue locations and how to register will be announced in coming newsletters and on the TREAT-NMD website.

FSHD registry launched in the UK
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A new registry for individuals with Facioscapulohumeral Muscular Dystrophy (FSHD) living in the United Kingdom has been launched. The registry, funded by the Muscular Dystrophy Campaign (MDC) and with support from TREAT-NMD, was launched earlier this month at a patient information day organised by the MDC held in Daventry. The registry has been very well received with over 150 patients registering in the last few weeks.

The new registry will allow clinicians and researchers to speed up the transition of treatments from the laboratory to the clinic by enabling faster recruitment for clinical trials and a better understanding of the condition.

As well as the internationally agreed core data (see ENMC workshop report Tawil et al, Neuromusc Dis, 20 (2010) 471-475) the registry collects information about pain, quality of life and experiences with the surgical procedure scapular fixation. All data is entered using a secure online system with patients entering the majority of data themselves. A nominated medical professional provides the required genetic information. For more information please visit the registry website www.fshd-registry.org/uk.

While this registry is intended for people based in the UK, there are a number of other national FSHD registries already available including in the Czech Republic, Italy and the United States whilst groups in France and the Netherlands are planning to launch registries in the coming months. Current and upcoming registries for FSHD are listed on the TREAT-NMD website.

Help support the work of the TREAT-NMD Alliance
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By supporting the work of the TREAT-NMD Alliance you can help us advance collaborative research with institutions across the world, which we believe will speed up the delivery of successful treatments. Your donation, regardless of size, would be gratefully received and would go to fund priority areas within the network, such as sustaining and developing patient registries and the development of outcome measures.

To donate, please visit our justgiving page which is overseen by Newcastle University, all donations from this page go directly and in full to the TREAT-NMD Alliance.

CMD care guidelines now available in different languages
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We are delighted to announce that family friendly care guidelines for congenital muscular dystrophy have been translated into French, German, Japanese, Latvian, Norwegian, Serbian, Spanish and Turkish and can now be downloaded from the CMD section of the TREAT-NMD website.

The translations are of the Management of Congenital Muscle Disease Family Guide which is a 40 page overview. The guide walks a family that is either new to a CMD diagnosis or has lived with CMD through the different physical, medical and functional aspects of the disease.

The guide is written in language that is easy to understand, includes an updated CMD nomenclature system that is gene based and has many candid photos of people with CMD.

We would like to take this opportunity to thank both Cure CMD and Muscular Dstrophy Canada for making this possible whilst also acknowledging the hard work that voluteers around the world.

Second newsletter from the Global FKRP Registry
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The Global FKRP Registry has just released its second newsletter and it is available for download from the Registry website in English and French, with German in progress. The newsletter is written for the benefit of patients and clinicians who participate in the Registry.

This year’s newsletter has focused on providing some preliminary analysis of the data held within the Registry, an update on current research, and relevant family conferences and events.

The Registry has now been active for just over two years and has around 300 patients registered from more than 30 countries around the world. The Registry website has recently been updated to make it easier for patients to register, with further updates on-going. The Global FKRP Registry Steering Committee also recently elected its first patient representative.

Workshop to provide consensus report on clinical effectiveness
of medicinal products for DMD and BMD
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A workshop with key stakeholders of the DMD and BMD community to look at ways to measure clinical effectiveness in the investigation of medicinal products for the treatment of DMD/BMD has been arranged for Friday 21st June in London.

This workshop aims to provide consensus to a report that TREAT-NMD will submit to the EMA in response to their current public consultation on the draft guideline which can be found here.

The topics to be discussed at the workshop include:

-  the natural history of DMD and BMD, what is clinically meaningful to patients and whether that can indeed be measured.
-  outcome measures and strength versus function and how these can be measured effectively.
-  biomarkers and the reliability of animal models.
-  strategies and design of clinical studies.

We would like to acknowledge that funding for this meeting has been provided by PPMD, UPPMD, AFM, Muscular Dystrophy Campaign and also the European Cooperation in Science and Technology Programme (COST).

Further details of the workshop will be available in due course. For further information please contact Stephen Lynn.

Registration is now open for Muscle Study Group 2013
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Registration for the Muscle Study Group 2013 Meeting is now open. The annual meeting will be held 16-18 September in Oxford, UK.

This year's meeting is entitled Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy.

Registration is limited so early registration is recommended and those who submit an abstract will get preference.

Meeting abstracts will be published in supplement form in the journal, Muscle and Nerve. Abstracts should be submitted by 31 May 2013 to Griggs_robertMSG@urmc.rochester.edu.

The Tentative Agenda and Abstract Submission Guidelines are available on the MSG website.

PPMD annual conference - registration closes 3rd June.
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Registration for the PPMD annual conference in Baltimore, Maryland closes on Monday 3rd June. The conference begins on 27th June and ends on 30th June. This is the largest, most comprehensive annual, international conference focused entirely on Duchenne muscular dystrophy.

Further details of the conference, accommodation and how to register can be found on the PPMD website.

Newcastle University includes cutting edge neuromuscular
diseases module as part of postgraduate degree programme
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From Autumn 2013 the MRC Centre for Neuromuscular Diseases at Newcastle will be organising a module entitled “Neuromuscular Diseases” as part of the one year Medical Sciences Masters in Research programme offered by Newcastle University (www.ncl.ac.uk/biomedicine/study/postgraduate/mres/). The module will cover:

1. How neuromuscular diseases impact on normal muscle structure and function, including normal muscle function and physiology, and the clinical and electrophysiological manifestations of muscle dysfunction across the neuromuscular system.
2. The molecular pathology of neuromuscular diseases, including the application of next generation sequencing and the development of gene and protein based diagnostics. Students will meet patients with neuromuscular diseases.
3. The assessment of therapeutic strategies through the preclinical modelling and assessment of neuromuscular diseases using cellular and animal systems, including critical appraisal of claims of therapeutic success in the preclinical setting.
4. The state of the art of novel therapeutic strategies for neuromuscular diseases including gene and cell-based therapies; understanding the targets and use of biomarkers; genetic and stem cell-based therapy for neuromuscular diseases; applications of antisense oligonucleotide technology in neuromuscular diseases; other targets for therapy development including downstream targets and protein upregulation. Moving studies into patients; the challenges of trial design in rare diseases.

The module forms part of the training programme in neuromuscular disease operated by the Centre and would particularly suit either clinical or basic scientists with a strong interest in neuromuscular diseases aiming to undertake research in this field. Further details, including entry criteria and how to apply can be obtained from the Newcastle University website. Additional enquiries should be addressed to Dr Steve Laval.

Research Director required at the European Neuromuscular Centre
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The European Neuromuscular Centre (ENMC) is seeking European based candidates for the position of the ENMC Research Director (0,1 FTE) whose main role is to chair the ENMC Research Committee and advise the ENMC Executive Committee to develop its strategic aims and objectives and their implementation.

The position will be available for an initial period of 2 years with the possibility of being extended for a further 2 years; starting date is 1 January 2014.

Closing date for appiications is 15 September 2013. For further information please visit the opportunities section of the website.

Job opportunity for clinical research associate in London
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A dynamic clinical research associate with experience in paediatrics and preferably neuromuscular disorders and in clinical trials with excellent communication skills and experience of working in a multidisciplinary team is required. This important post will be based in the Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, in London.

Primary focus will be on the clinical trials and natural history studies on Duchenne muscular dystrophy and spinal muscular atrophy in which the centre is particularly active in. The successful candidate will be able to interact with the team of research scientists working on preclinical optimisation and development of novel therapeutic interventions, with the clinical team of the Dubowitz Neuromuscular Centre, one of the largest Paediatric Neuromuscular Centres in Europe, and with the excellent team of the GOSH Clinical research Facility. The candidate will be part of a smaller team within the Centre, composed of study coordinators, research nurses, research therapists and other clinical research fellows.

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31st May 2013
TREAT-NMD newsletter - 31st May 2013
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