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16th November 2011
TREAT-NMD conference report

Last week's TREAT-NMD international conference in Geneva brought together more than 250 delegates with an interest in neuromuscular disease: patients, families, clinicians, basic scientists, industry representatives and other key experts. For those who could not attend, a full conference report now published on the TREAT-NMD website provides an overview of the sessions plus links to the abstracts provided by all speakers. The high calibre of the speakers and the interactive nature of the event provided a revealing insight into the current state of translational research in the neuromuscular field and its future promise and challenges.

Recommendations for Centres of Expertise adopted unanimously
by the European Union Committee of Experts on Rare Diseases
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On 24 October, the third meeting of the European Union Committee of Experts on Rare Diseases (EUCERD) unanimously adopted the Recommendations on Quality Criteria for Centres of Expertise for Rare Diseases in Member States (pdf). With 51 members, representing all 27 Member States of the EU, the panel covers all areas relevant to the fields of rare diseases and orphan drugs including academia, government, the biopharmaceutical industry and patient organisations.

Centres of Expertise and European Reference Networks for rare diseases were proposed in order to help organise care for large number of rare conditions affecting scattered patient populations across Europe. This first set of recommendations adopted by the EUCERD committee elaborate quality criteria which Member States can incorporate into their national processes to designate Centres of Expertise.

In common with other areas of rare disease research, the neuromuscular field is able use the quality criteria defined in the EUCERD recommendations to inform planning for field-specific Centres of Expertise. The CARE-NMD project, which aims to help establish a European network of reference centres to improve the quality of care for Duchenne muscular dystrophy (DMD), is an example of where the neuromuscular field will benefit from the groundwork being done at a pan-European rare disease level.

Postdoctoral Researcher Opportunity
RNA Interference & Gene Therapy for Muscular Dystrophy
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The Royal Holloway in London, UK is looking to appoint a post-doctoral scientist with excellent skills in molecular biology, aseptic cell culture, and molecular cloning. Experience in muscle biology and histology, RNAi/ shRNA gene expression knock-down, adeno-associated virus vector (AAV) gene transfer, and in vivo experimentation would be an advantage but training is available.

The successful applicant will join a leading international research team evaluating a revolutionary new approach to combat oculopharyngeal muscular dystrophy (a triplet repeat autosomal dominant muscle disease) in conjunction with the Sydney-based biotechnology company, Benitec PLC, and the Institut de Myologie in Paris. He/ she will be responsible for developing and testing shRNA and gene therapy systems to modulate aberrant gene expression and to counter muscle atrophy. Post is available initially for 18 months with potential for further extension towards a clinical trial.

Download further details or visit the Royal Holloway website to begin the application process.

Update on the Iranian patient registry for DMD
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The creation of the Duchenne / Becker registry in Iran, which collaborates with the TREAT-NMD global DMD registry, was made possible thanks to the initial support of the Iranian Molecular Medicine Network and subsequently the Genetics Department of Social Welfare and the Ministry of Health.

Data gathered in the registry will provide medical experts with both genetic and medical information. A new website has recently been launched and patient information is currently being added. The family guide to the standards of care for DMD is currently being translated into Persian. Once complete this will be available for download from both the Iranian registry website and the TREAT-NMD website. Click the "more" link below to visit the registry website.

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16th November 2011
TREAT-NMD newsletter - 16th November 2011
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