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25th April 2012
Message from Annemieke and Hanns

Dear colleagues and friends,

We are delighted to announce that we have been elected by the Executive Committee as its Chair and Vice-Chair. We are looking forward to working with all of you to ensure a smooth transition as TREAT-NMD moves into this new phase of collaboration.

A new three-year action plan has been drafted to define collaborative tasks and objectives that the neuromuscular community should achieve from 2012 to 2015, and you can find further details of the planned work in the news item below. Thanks to the combined efforts of many in the field, some of this work has already started, and we invite all of you to participate in those parts of the programme that are of interest to you.

We are also extremely excited by the positive evaluation of several applications to the recent European Union FP7 HEALTH call which will have a major impact on the neuromuscular and rare disease fields – see the article later in this newsletter. The active input and contributions of stakeholders worldwide will remain essential to TREAT-NMD's future and we encourage you to contact us directly with your thoughts and suggestions.

Hanns Lochmüller (Chair)             Annemieke Aartsma-Rus (Vice-Chair)

TREAT-NMD three-year action plan
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During its EU-funded period, the partners within TREAT-NMD were obliged to work to a clearly defined "description of work", with agreed deliverables, milestones and expected achievements. This milestone-driven approach clearly drove the network's progress forward and it was therefore considered crucial to maintain this momentum by establishing new goals and defining the priorities and direction for future collaborative work across the NMD field.

Led by the TREAT-NMD Academic Task Force, TREAT-NMD participants across the world have drafted a new Description of Work or "action plan" for the next three years, built around the network’s core tools and resources. Clearly defined tasks and goals have been set out and task participants identified. The achievement of these will require funding, which in a number of cases has already been secured. Meanwhile, having a document that clearly defines a new set of priorities and tasks for each activity should help participants who still need to source funding to do so. As well as the tasks described in the Description of Work, additional projects led by network participants will make use of the network tools. Anyone interested in contributing to these tasks or proposing new areas of activity for the network is invited to contact the task leaders named. Click the "more" link to see a summary of all activities.

Results of the recent FP7 HEALTH call:
Success for neuromuscular and rare disease projects
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The European Commission made rare diseases one of its priorities for funding in 2012, with a major financial commitment to rare disease topics contributing towards the new International Rare Disease Research Consortium (IRDiRC) goals. Several groups with a neuromuscular interest took up the challenge of submitting applications to the FP7-HEALTH-2012-INNOVATION-1 call, and last week applicants were notified of the success of their proposals. We are delighted to report that a number of proposals involving TREAT-NMD participants have been invited to begin negotiations towards the award of a grant.

The following applications have now been invited to begin negotiations.

Neuromics (Coordinator: Olaf Riess, Tübingen, Germany, Co-coordinators: Gert-Jan van Ommen, Brunhilde Wirth): an integrated European project on omics research in rare neuromuscular and neurodegenerative diseases.
Submitted under: HEALTH.2012.2.1.1-1-B: Clinical utility of -Omics for better diagnosis of rare diseases

RD-Connect (Coordinator: Hanns Lochmüller, Newcastle, UK): an integrated platform connecting registries, biobanks and clinical bioinformatics for rare disease research.
Submitted under: HEALTH.2012.2.1.1-1-C: databases, biobanks and ‘clinical bio-informatics’ hub for rare diseases

SUPPORT-IRDiRC (Coordinator: Ségolène Aymé, Paris, France): Support for international rare disease research to serve the IRDiRC objectives.
Submitted under: HEALTH.2012.2.1.1-1-A: Support for international rare disease research

SKIP-NMD (Coordinator: Francesco Muntoni, London, UK): Phase I/IIa clinical trial in Duchenne muscular dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53.
Submitted under: HEALTH.2012.1.4-4. Targeted nucleic acid delivery as an innovative therapeutic prophylactic approach

OPTIMISTIC (Coordinator: Baziel van Engelen, Nijmegen, Netherlands): an observational prolonged trial in myotonic dystrophy type 1 to improve QoL standards, a target identification collaboration.
Submitted under: HEALTH.2012.2.4.4-2: Observational trials in rare diseases

RARE-bestpractices (Coordinator: Domenica Taruscio, Rome, Italy): a platform for sharing best practices for management of rare diseases.
Submitted under: HEALTH.2012.2.4.4-3: Best practice and knowledge sharing in the clinical management of rare diseases

Click the "more" link below for further information about each of these proposals.

An update on neuromuscular developments in Hungary
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Hungarian TREAT-NMD partners have been directly involved in three highly significant developments in the neuromuscular field in Hungary this month: the establishment of a National Neuromuscular Centre, a presentation to parliament, and the first CARE-NMD workshop on DMD care.

An official agreement was signed on Monday 16th April which formally established in Budapest the first specialist Hungarian Neuromuscular Centre. Although collaboration has been on-going for some time, the agreement - between the Hungarian National Institute for Environmental Health (NIEH), Semmelweis University 2nd Paediatric Clinic, Szent Janos Hospital and Northern-Buda Corporated Hospitals - Rehabilitation Unit, National Institute of Children's Health, Misko Foundation and the Healing Goodwill Foundation for Children with Neuromuscular Disorders - provides a formal basis for future cooperation. As the first specialist centre in the country, it will also form part of a network of expert centres to be developed over time in line with European recommendations on Reference Networks; it is hoped that a second centre in Szeged will be inaugurated soon.

Postdoctoral position available at the Institute of Myology
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A postdoctoral position in Gisèle Bonne's team as part of the Cure-CMD funded project “Trans‐Splicing for L‐CMD” is now available.  This position will set up and evaluate gene therapy for LMNA‐related Congenital Muscular Dystrophy (L‐CMD) by trans‐splicing approach. Gisèle's team uses knock‐in mouse models and primary cell culture to study in vivo and ex vivo muscle and heart development and function and test therapeutic approaches.

Further details can be found by clicking 'more'

Register now for the WMS pre-congress training course in Perth
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Places for the pre-congress training course are filling up fast. Scheduled for Monday 8th October in Perth Australia, the emphasis of this course will be on the clinical approach of patients who present with symptoms of weakness and the interpretation of the muscle biopsy. The course lecturers are internationally well-renowned experts in the field and have extensive experience in the evaluation, diagnosis and treatment of neuromuscular diseases. 

Don't forget early bird registration for the World Muscle Congress itself closes on 30th April.

Further details of the satellite course and how to register for the congress itself can be found on the congress website.

Registration being taken for PPMD Connect conference
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Registration is open for PPMD's Connect conference. The conference will be held from 28th June - 1st July in Fort Lauderdale, Florida. Early bird registration for this conference ends on 21st May.

This year PPMD will be hosting a three part series on Clinical Trials. There will be panel presentations from parents whose children are or were participating in trials, from clinicians who run the trials and from industry leaders themselves who develop the trials. The interactive panels will provide a glimpse into the future of Duchenne trials.

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

Update in Neuromuscular Disorders Course 2012
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Registration is now open for the Update in Neuromuscular Disorders course which will be held 8th - 11th May 2012 in London, UK.

This established paediatric and adult course is run jointly between the Dubowitz Neuromuscular Centre, UCL Institute of Child Health and the MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology,  and is now in its fifth year. RCP and RCPCH CPD approval has been applied for.

Please visit the course website for program and registration details.

Next Assessment Round for ENMC Workshop Applications
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The deadline for sending in applications for ENMC workshops to be conducted in the first half of 2013, is 1st September 2012.  Application forms for a workshop can be downloaded from the ENMC website.

Please be informed that incomplete applications and applications submitted after the deadline, will not be processed.

If you have any questions regarding this, please do not hesitate to contact the ENMC office.

Submission deadline 1st September 2012.

First call for EPNS Researchers Meeting
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The European Paediatric Neurology Society's Researcher Meeting has been scheduled for 14th & 15th December 2012 in Rheinfelden, Germany close to Basel.

The main topics for talks are epilepsy and ataxia. Whilst the following research groups will also form part of the meeting, neurometabolic diseases, autoimmune-inflammatory-diseases, neonatal neurology, neurodegeneration and neuroprotection, epilepsy, genetics, developmental neurology, ataxia and neuromuscular diseases.

If you wish to express an interest in participating in the neuromuscular disease research group please contact one the following research heads Thomas Sejersen, Nathalie Goemans, Markus Schülke or Volker Straub.

Please visit the EPNS website for further details of this meeting.

Have you anything to tell us?
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Don't forget you can submit an article or details of a conference, meeting or event via the TREAT-NMD website.

Our newsletter is sent out on the last Wednesday of every month, your submission needs to be received the week before that to be considered for inclusion.

Please visit the 'submit site updates section' of the site to get in touch.

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25th April 2012
TREAT-NMD newsletter - 25th April 2012
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