28th March 2012
Translators required for congenital muscle disease family guide

The Management of Congenital Muscle Disease Family Guide is a 40 page overview that 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.

Cure CMD are currently soliciting volunteer translators who can help them translate this guide into their native language. All translations will be posted on the TREAT-NMD and Cure CMD websites.

If you are interested in translating the guide, please contact Diane Smith-Hoban

Paris Summer School of Myology 2012
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This year's Paris Summer School of Myology has been scheduled for 21st-29th June. Delivered in English, this seven-day intensive course is open to students worldwide, however places are limited to 60.

Topics covered include basic myology, muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital muscular dystrophies, congenital myopathies, metabolic myopathies, myasthenic syndromes, chanelopathies, cardiomyopathies along with management and therapies.

Further details of the course itself and how to apply can be found on the Institut de Myologie website.

MyoGrad Berlin Summer School for Basic Muscle Science 2012
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The Muscle Science Summer School in Berlin will run from 18th-22nd June and covers the whole field of basic muscle research. Some of the world's most highly recognized experts in their respective fields will give lectures on muscle contraction, muscle development, muscle metabolism, neuromuscular transmission, and novel experimental treatment strategies.

Please visit the MyoGrad website for details of this year's program and how to register. The registration deadline is 31st May 2012.

Hungarian standards of care workshop for DMD
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Places are still available for this free one day training course which will be held on Wednesday 18th April in Budapest Hungary.

Delivered in both Hungarian and English, the course includes interactive discussion sessions for participants relating to various aspects of DMD care. This is a great opportunity for attendees to engage with international DMD experts and other clinicians in an informal environment.

This professional training event has been organised in collaboration with our local CARE-NMD Partner, the National Institute for Environmental Health (NIEH), and Heim Pal Childrens' Hospital.

Speakers & topics include:

  • Veronika Karcagi PhD (CARE-NMD Hungarian Partner)
    Head of the Dept. of Molecular Genetics and Diagnostics at the National Institute of Environmental Health, Budapest, Hungary

    Differential diagnosis of DMD based on molecular analyses

  • Janbernd Kirschner MD, PhD (CARE-NMD Project Leader)
    Associate professor, paediatric neurologist, University Medical Center, Freiburg, Germany

    Overview of the CARE-NMD project

  • Ágnes Herczegfalvi PhD (CARE-NMD Hungarian Partner)
    Head of the Dept. of Neurology, Paediatric Clinic II., Semmelweis University, Budapest, Hungary

    The current state of DMD patient care in Hungary / Duchenne betegek ellátásának hazai helyzete

  • Dr. Márta Garami (CARE-NMD Hungarian Partner)
    Database Manager, Dept. of Molecular Genetics and Diagnostics at the National Institute of Environmental Health, Budapest, Hungary

    National and international patient registry for Duchenne patients / Nemzeti és nemzetközi betegregisztráció Duchenne betegek részére

  • Thomas Sejersen MD, PhD (CARE-NMD Partner)
    Associate Professor, Paediatric Neurologist, Head of Clinical and Research team for Neuromuscular Disorders in Childhood, Astrid Lindgrens Barnsjukhus - Karolinska Institute, Stockholm, Sweden

    Standard of care for DMD with special focus on heart and respiration & Clinical trials and future therapies in DMD

  • Enrico Bertini MD, PhD
    Neurologist, Unit leader of Molecular Medicine, Division of Neurology, Children's Hospital "Bambino Gesú", Rome, Italy

    Outcome measures in DMD

  • Birgit F. Steffensen, PT, PhD (CARE-NMD Partner)
    Senior Researcher, Rehabiliterings Center for Muskelsvind, Arhus, Denmark

    Rehabilitation and psychosocial aspects in DMD care
Abstract deadline approaches for AOMC annual meeting
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The 11th Scientific meeting of the Asian and Oceanian Myology Center (AOMC) will be held on 7th - 8th June 2012 in Kyoto Japan.

Abstract submission deadline is 15th April with early bird registration available until 30th April.

At the Kyoto meeting there will be two sessions on metabolic myopathies. The first is lipid storage myopathy, commonly seen in China, and a disease treatable with riboflavin in many patients. The second session will be on glycogen storage diseases, especially Pompe disease, which is now treatable with enzyme replacement therapy.

Dutch Duchenne Parent Project call for research proposals:
deadline 1st May
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The Dutch Duchenne Parent Project NL (DPP NL) stimulates research in the search for a cure for Duchenne Muscular Dystrophy. DPP NL sponsors promising research and fellowships, faster exchange of data and international collaboration.

Researchers can apply for funding for their research projects via the DPP NL website and applications need to be received by 1st May. Results will be announced 1st October.

For more information about the application procedure visit the Parent Project website.

Conditions for funding:

- Selection by international scientific advisory board
- Innovative research for finding a cure
- Avoidance of diarchy in research
- Multi-track policy: the widest possible range of research should be covered as it is not possible to say which research will lead to the ultimate solution.
- Results should be generally available and measurable
- Selected projects will be monitored closely during their progress and researchers are required to submit progress reports
- Based on the results the scientific advisory board advises the Duchenne Parent Project annually about the continuation of sponsorship on the ongoing projects

Further information on the application procedure: dpp@euronet.nl

4th Meeting of the European Union Committee of Experts on Rare Diseases
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The fourth meeting of the European Union Committee of Experts on Rare Diseases (EUCERD) took place on 26th & 27th January 2012 in Luxembourg. The plenary was preceded by preparatory meetings by stakeholder groups, which resulted in fruitful discussions concerning the two main issues for discussion, the Clinical Added Value of Orphan Drugs (CAVOD) and European Reference Networks. Further details can be downloaded from the EUCERD website.

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28th March 2012
TREAT-NMD newsletter - 28th March 2012