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6th September 2013
TREAT-NMD Alliance Conference 2013 – deadline for registration is approaching

We are delighted by the number of delegates from across the world who have already signed up to ensure their place at the TREAT-NMD Alliance Conference in Newcastle at the end of October.

Places are limited so anyone wishing to attend the TREAT-NMD Alliance Conference is strongly encouraged to register as soon as possible. Registration for the conference is free of charge for academics, TREAT-NMD-affiliated registry curators, patients and patient representatives. The online registration, which takes only a few minutes to complete, will close on Monday 30th September 2013.

The conference focuses on:

-  results and achievements of TREAT-NMD related research
-  patient involvement and ethics in TREAT-NMD related research
-  upcoming new themes of collaborative research

Satellite meetings include a CARE-NMD project meeting, TREAT-NMD Global Database Oversight Committee (TGDOC) meeting, TREAT-NMD Alliance Executive Committee meeting and the TREAT-NMD Advisory Committee for Therapeutics (TACT) meeting.

Further information about the programme, travel and accommodation options, and how to register can be found on the TREAT-NMD website.

Don’t Stop Research on Rare Disease: sign a petition to the
European Parliament
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The rare disease community has expressed major concerns about a number proposed amendments to the EU Data Protection Regulation released by Jan Philipp Albrecht at the beginning of 2013 (Enabling data sharing: two publications highlight ethical and legal questions to be addressed. RD-Connect Newsletter. 25 June 2013). The declared focus of the reform of the Data Protection Regulation is to strengthen privacy protection and personal control over data in general, but according to researchers, using the same rules for health research could have serious consequences for rare diseases.

It has been suggested that the new Data Protection Regulation reforms will hinder data sharing amongst researchers by preventing the sharing of “codified” patient data. This will have major consequences for research conducted by biobanks and registries which require participation across national borders to gain statistically meaningful results.

The European Platform for Rare Disease Registries (EPIRARE) together with the European Organisation for Rare Diseases (EURORDIS) have succeeded in engaging Members of the EU parliament in a debate “Data protection: potential impact of the revision of the current legislation on medical research in the rare diseases field” in the hope to express their concerns and prevent reforms from passing through Parliament. During the debate EPIRARE and EURORDIS intend to present the results of their petition “Don’t Stop Research on Rare Diseases” which has reached a sizable number of more than 2700 signatures but still a long way off the target of 10000. The meeting has been scheduled for 26th September 2013 in the EU Parliament in Brussels and the petition will remain open until this date. Click 'more' for further details.

New publication highlights the utility of the DMD patient registries
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A recent paper published in Human Mutation, The TREAT-NMD Duchenne muscular dystrophy registries: conception, design and utilisation by industry and academia, describes in detail how the TREAT-NMD national patient registries for DMD were established. It goes on to show how the registries have grown since their inception in 2007, and further highlights their success in fostering collaboration between academia, patient organisations and industry ultimately leading to increased clinical trial opportunities for patients and translating directly to improvements in patient care and treatment options.

This publication is the first in a series of publications currently in preparation analysing genetic and clinical data in DMD and can be found in the DMD section of the TREAT-NMD website.

TREAT-NMD Alliance submits response to the EMA
public consultation on DMD/BMD
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In March 2013, the EMA published a draft guideline on the clinical investigation of medicinal products for DMD/BMD and invited comments during a public consultation phase. As part of the coordinated response to the public consultation, the TREAT-NMD Alliance organised a workshop in June 2013 at The Wellcome Trust head office in London to discuss many of the issues in the draft guideline, such as natural history of the disease, methods to assess efficacy, use of animal models and strategies and design of clinical studies. The workshop agenda and publicly available presentations can be found on the TREAT-NMD website here.

We are delighted to inform you that the TREAT-NMD Alliance has now finalised and submitted to the EMA its response to the public consultation, the deadline of which was 31st August 2013. The final version of the submitted response to the public consultation can be found on the TREAT-NMD website here.

We would like to acknowledge the support provided for the workshop and thank all those that contributed to the workshop and provided input to the TREAT-NMD Alliance submission to the public consultation.

DMD research overview now available in Chinese
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The research overview section for DMD on the TREAT-NMD website is regualrly updated by Annemieke Aartsma-Rus and keeps those affected by DMD up to date with the latest information from the research field. Written to be understandable to those without a medical background, the overview looks at current clinical trials in DMD and explains the different research approaches.

This section has now been translated into Chinese by Prof Haifang Yin and her group at the Research Center of Basic Medical Science of Tianjin Medical University. We would like to thank Haifang and her team for all their hard work and hope that their translation will help the Chinese speaking DMD community thoughout the world to understand the research that is currently being undertaken.

SMA Guide now available in Spanish
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We are delighted to announce that the spinal muscular atrophy guide for families and doctors has been translated into Spanish and is available to download from the TREAT-NMD website.

TREAT-NMD worked with the authors of the international consensus statement on care for patients with spinal muscular atrophy to create useful summary factsheets based on the full published document. The guide is currently available in sixteen different languages and has been downloaded approximately 500 times from our website in the last year alone.

We would like to take this opportunity to thank Dr. Claudia Castiglioni from the Neurologist of Clinica Las Condes in Chile and Families of SMA also from Chile for their work with this guide. We are sure this translation will be a great benefit to Spanish speakers affected by SMA. If you are interested in voluntarily translating the guide into a language we currently don't have, please visit the TREAT-NMD website for further details.

IORMC 2013 - abstract deadline approaches
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The abstract submission deadline form the third Indian Ocean Rim Muscle Colloquium (IORMC) is 30th September. This year's colloquium will be held in Singapore from 12th -13th December 2013. The brain-child of Miranda Grounds of the University of Western Australia, this meeting brings together leading muscle researchers from India, Singapore and Australia with the aim of developing collaborations in the Indian Ocean region.

Previous meetings were extremely successful and catalysed multiple interactions and research collaborations between researchers from across the region. The excellent travel links to Singapore are hoped to help with extending participation further to other 'rim' countries including South Africa and Thailand. The Colloquium is open to PhD students and post-docs as well as established investigators and provides a great opportunity for those at the early stages of their research careers to present their work to a specialist audience. Please visit the IORMC website for further details

TREAT-NMD Alliance Conference
Past newsletters
6th September 2013
TREAT-NMD newsletter - 6th September 2013
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