25th January 2012
The TREAT-NMD Alliance: Current and Future Plans for 2012 and Beyond

In the last newsletter of 2011, we talked about how collaboration and networking over the last five years has supported the various activities of TREAT-NMD. With the formation of the TREAT-NMD Alliance, it is hoped that this collaborative approach will continue.

This will be coordinated by a 12 member Executive Committee with 9 academic members (scientists and clinicians) and 3 members from patient organisations. So far, elections have been held for the academic members of the Executive Committee and out of 21 nominees, the following nine people have been elected:

-  Annemieke Aartsma-Rus (Leiden University Medical Centre, NL)
-  Kevin Flanigan (Nationwide Children’s Hospital, Ohio, USA)
-  Eric Hoffman (Children’s National Medical Centre, Washington DC, USA)
-  Jan Kirschner (University Medical Centre Freiburg, Germany)
-  Hanns Lochmuller (Newcastle University, UK)
-  Eugenio Mercuri (Catholic University, Rome, Italy)
-  Ichizo Nishino (National Centre of Neurology and Psychiatry, Japan)
-  Kathy North (University of Sydney, Australia)
-  Thomas Sejersen (Karolinska Institute, Sweden)

On behalf of the TREAT-NMD Alliance, we would like to acknowledge the help and support from the academic Task Force who elected the academic members of the Executive Committee. Currently, the elections for the patient organisation members of the Executive Committee are ongoing with 17 nominations from patient organisations around the world, and we look forward to informing you of the outcome of this election in our next newsletter on the 29th February (which is also Rare Disease Day!).

Following the patient organisation election the first full 12-member Executive Committee, who will be tasked with providing ongoing oversight and leadership to the TREAT-NMD Alliance, will work with our many collaborators and supporters to continue the activities of TREAT-NMD. Newcastle University has committed to providing ongoing secretariat support to the Executive Committee and the TREAT-NMD Alliance and we will continue to provide updates on progress via the TREAT-NMD newsletter.

The first duties for the new Executive Committee will be to revise and approve the current versions of the Members’ Charter, as well as the charter that describes the new governance and oversight structure for the TREAT-NMD Alliance. From 2007 to the end of 2011, leadership of TREAT-NMD was provided through the Governing Board that consisted of representatives from the organisations who received funding from the European Commission via the FP6 grant. The new Executive Committee will define the TREAT-NMD Alliance governance model as well as any advisory or sub-committees necessary to help ensure the Alliance is properly managed from 2012 onwards.

The TREAT-NMD web site and newsletter will continue to provide ongoing information on the activities of the Alliance and the Executive Committee. The Executive Committee look forward to working with you all on developing the TREAT-NMD Alliance and our continuing collaboration!

Family Guide for CMD now available to download
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The congenital muscular dystrophy (CMD) family guide is now available to download from the TREAT-NMD website. The guide summarizes an international consensus on diagnosis and medical care. Created by a working group which was lead by Cure CMD the effort was also supported by AFM-Association Française contre les Myopathies, Telethon Italy and TREAT-NMD.

The family-guide "translates" the full academic publication published in November 2010 into a form that is more readily understandable to non-specialists and will enable families to have a clear guideline that they can use to discuss their care with their care providers.

Click on the 'more' link to go to the CMD section of our website to download the guide.

GSK responses to patient questions now available in Japanese!
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Many patients and families around the world are aware that clinical trials in neuromuscular diseases are now running and seek information on whether they can get involved.

TREAT-NMD regularly receives questions from patients and families anxious to find out which trials are happening, which clinics are involved, and which patients might be eligible.

In early 2011 we put these questions to pharmaceutical companies running trials. GSK who have a program in DMD replied and we published their responses on the TREAT-NMD website and in our newsletter. These responses have since been translated into Japanese and have been published on Japan’s Registry of Muscular Dystrophy website Remudy.

Are you a Friend of Rare Disease Day?
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29th February marks the fifth international Rare Disease Day coordinated by EURORDIS and organised with rare disease national alliances in 25 European countries. On this day hundreds of patient organisations from more than 40 countries worldwide are organising awareness-raising activities converging around the slogan “Rare but strong together”.

To increase awareness of the Rare Disease Day why not consider becoming a "Friend of Rare Disease Day"? Friends can be researchers, health professionals, care givers, public authorities, industry, individual patients, patient groups and any other person or entity genuinely interested in improving the lives of people affected by rare diseases. Being a “Friend” requires limited commitment to the Rare Disease Day campaign and acknowledges the fact that not everyone can give the same level of time and effort.

There's still time to get involved - click 'more'!

ECRD 2012 Abstract deadline extended
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The poster abstract submission deadline for European Conference on Rare Diseases & Orphan Products - ECRD has been extended to 31 January 2012.

ECRD 2012 will be held at the Management Centre Europe (MCE) Conference Centre in Brussels, Belgium from 23 - 25 May 2012.

The ECRD Conference is the unique platform/forum across all rare diseases, across all European countries, bringing together all stakeholders - academics, health care professionals, industry, policy makers, patients’ representatives.

This is an annual event, providing the state-of-the-art of the rare disease environment, monitoring and benchmarking initiatives.

Covering research, development of new treatments, health care, social care, information, public health and support at European, national and regional levels this year's themes that run throughout the conference are:

  • Centres of Expertise and European Reference Networks
  • Information & Public Health
  • Research from Bench to Bedside
  • Orphan Products & Rare Disease Therapies
  • Patients’ Empowerment

For further details about the conference itself or to see the preliminary programme visit: www.rare-diseases.eu. Poster abstracts can be submitted at: www.rare-diseases.eu/2012/Submit-a-Poster.

AFM Call for proposals
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The deadline for the second session of the AFM call for prosopals for 2012 is 9th March. The international call aims to support research which will:

-  Increase understanding of the neuromuscular system
-  Improve care and quality of life of patients with neuromuscular diseases
-  Encourage the development of therapies for neuromuscular diseases and rare genetic diseases

Types of financing include:

- One year trampoline grants
- PhD fellowships for one year, renewable for a second and third year
- Post doctoral fellowships for one year, renewable for a second year
- Research projects for one year, renewable for a second year, and exceptionally for a third year
- Ambitious research projects with PhD or post-doctoral fellowships will be recognised as an AFM-sponsored program

Applicants will be notified in mid-July 2012.

Please download the PDF for further details or visit the Institut de Mylogie website.

World Muscle Society Congress update
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The World Muscle Society Annual Congress will take place in Perth, Australia this year on 9th-13th October 2012. The annual meeting brings together world leaders in clinical, pathological and scientific research into neuromuscular disorders. This is the first time the Congress will be held in Australasia enabling delegates to experience Australian hospitality and culture at first hand.

This is an exciting time in the neuromuscular disorders field, with next generation sequencing techniques starting to have a huge impact in novel disease gene discovery and approaching use in molecular diagnostics and potential therapies for these conditions, based on understanding the pathobiology of the diseases, in clinical trials.

The four chosen themes for the congress in Perth are:

-  Impact of New Diagnostic Techniques on Clinical Practice in Neuromuscular Disorders
-  Congenital Myopathies
-  Standards of Care in Neuromuscular Disorders (inc. cardiac, respiratory, orthopedic, swallowing and feeding, physical medicine/therapy and rehabilitation).
-  Advances in Therapy for Neuromuscular Disorders

Abstract submission deadline is 31st March and early bird registration ends 30th April.

25th January 2012
TREAT-NMD newsletter - 25th January 2012