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26th July 2013
Register now for the TREAT-NMD Alliance meeting to secure your place

Registration is well underway for the upcoming TREAT-NMD Alliance meeting in Newcastle. We are delighted by the number of delegates from Australia, USA, Japan and also right across Europe who have already signed up to ensure their attendance in October.

This will be 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 areas of research and collaboration. Attending delegates will learn about recent studies of MRI as an outcome measure, next generation sequencing of neuromuscular diseases as well as emerging therapies for CMT, SMA and myotonic dystrophy.

Further information about the programme and how to register can be found on the website. Anyone wishing to attend this meeting is strongly encouraged to register sooner rather than later.

Egyptian NMD registry goes live!
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The past month has seen several exciting developments in the neuromuscular field in Egypt, including access to a specialised neuromuscular clinic, the official launch of the Egyptian NMD Registry, and the translation of the CARE-NMD website into Arabic.

A specialised muscle clinic service is now available at the Egypt Air Hospital in Cairo, and is able to see children and adults with muscle conditions for clinical and genetic diagnosis. The clinic is run by Dr Rasha El Sherif who also curates the recently launched Egyptian Neuromuscular Disease Registry.

The registry is open for patients with a confirmed DMD/BMD or FSHD diagnosis in Egypt, and is also able to accept registrations from patients with these conditions in other Arab countries where a local registry does not exist. A key aim of the registry is to improve understanding and care for neuromuscular diseases. For further information on the clinic or the registry, please contact Dr Rasha El Sherif.

The CARE-NMD website has also been translated into Arabic, making information about the project available to interested parties across the Middle East. A translation of the DMD Family Guide into Arabic is in progress and will be made available once complete.

Dr Rasha El Sherif would be delighted to hear from clinicians, physiotherapists and other health professionals working with neuromuscular patients in Egypt and the wider region.

Publication of the 2013 report
‘State of the Art of Rare Disease Activities in Europe’
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The neuromuscular field has organised itself to work collaboratively and effectively across borders, with the shared goal of providing a higher quality of care and greater prospects for patients worldwide. For those seeking to view this success against a wider rare disease backdrop, the European Union Committee of Experts on Rare Diseases (EUCERD) has recently published a comprehensive report designed to raise awareness of European activities across the rare disease field broadly. The report is produced annually by the ‘EUCERD Joint Action: Working for Rare Diseases’ and is designed as a one-stop-shop for information on European rare disease activities up to the end of 2012. The final result is the product of extensive multi-stakeholder input across all EU Member States, and is divided into five volumes to help readers find exactly what they need, quickly.  For instance, there is a volume dedicated to the most recent activities of the European Commission, and another focusing on the developments in the world of orphan medicinal products and the European Medicines Agency.

Volume 1 is particularly popular, providing a solid overview of rare disease activity in Europe to date, whilst for previous readers of the State of the Art, volume 2 details all new developments since last year’s report. If you need information about the current state of rare disease activity in a particular Member State, volume 5 is indispensable, as it presents country-by-county updates and insights. In short, the State of the Art report is an excellent resource for those seeking information on any of the following: the development of centres of expertise; registries; genetic testing resources and activities; patient organisation activities; information resources; guidelines and recommendations; educational initiatives; research and funding mechanisms and participation in EU-level projects; rare disease conferences and events; orphan medicinal product incentives, availability, reimbursement and pricing policies; and specialised social services. All volumes of the report are now available to download from the EUCERD website.

Keep up-to-date with the latest developments and therapies in DMD
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To help patients and families keep abreast of the latest developments and therapeutic approaches for DMD, Dr. Annemieke Aartsma-Rus (Leiden University Medical Center), the Vice-Chair of the TREAT-NMD Executive Committee, has reviewed and updated her DMD research pages to provide information about the different therapeutic approaches currently currently in development.

These pages can be found in the dedicated DMD section of the TREAT-NMD website. Here Annemieke describes the advantages and disadvantages of each approach and lists the hurdles that have to be overcome before these approaches can be applied to patients. This section is an extremely valuable resource for those affected by DMD as it provides the information on these complex therapies in a more understandable form.

International summer school and workshop on rare disease
and orphan drug registries - last call!
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The Epirare project focusses on the development, sustainability and impact of patient registries throughout the EU have two events organized for the coming months.

Firstly, an international summer school entitled “Rare Disease and Orphan Drug Registries” will be held in Rome, 16-20 September 2013. This course will take participants through the main concepts and practical steps that must be undertaken in the establishment and management of a rare disease registry to ensure its usefulness, soundness and sustainability. Applications to attend this course need to be submitted by 30 July.

Secondly, a workshop in Rome, 21-22 October 2013, will allow attendees to share rare disease registry experiences, pinpoint their strengths and weaknesses whilst also providing an excellent opportunity for networking. Details of this second workshop are available on the Epirare website, regisitration and abstract submission closes 31 July.

Date change for Update on Neuromuscular
Diseases Course in 2014
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The date of the neuromuscular update course in 2014 has recently been changed to 17-20 March.

This established course is organised by Professor Francesco Muntoni, Dr Adnan Manzur, Dr Stephanie Robb, Professor Mary Reilly and Professor Michael Hanna. Now in its seventh year the course is aimed at anyone with an interest in neuromuscular disease, either paediatric or adult.

Registration for the course in London UK, begins in November 2013. Early registration is recommended to avoid disappointment.

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 applications is 15 September 2013. For further information please visit the opportunities section of the website.

TREAT-NMD newsletter - summer break
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This is our final newsletter before we take our traditional summer break and we would like to take this opportunity to thank everyone who has been involved with the newsletter throughout the last year.

We will return to your inboxes once again in September with news and information from the neuromuscular field. If you have any news or information that you would like to be considered for inclusion in forthcoming newsletters, you can submit your article here.

We hope that those of you with vacations coming up have a great break and we look forward to contacting you all again in September.

TREAT-NMD Alliance Meeting
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26th July 2013
TREAT-NMD newsletter - 26th July 2013
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