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28th January 2011
New CARE-NMD website launched

CARE-NMD is a 3-year project to implement best-practice standards of care for Duchenne muscular dystrophy (DMD) across Europe. A key aim of the project is to make high quality information on Duchenne care available to as many patients, families and clinicians as possible.

The project has today launched a revised project website, with information available in the seven languages of the Associated Partners: Bulgarian, Czech, Danish, English, German, Hungarian and Polish. Translations of the Family Guide are complete or ongoing in all seven languages, and where complete are available to download. Over the lifetime of the project, the website will be further expanded by the project partners, with additional languages and localised information. This will cover a range of topics associated with care for DMD, including information on patient organisations, details of clinical training events, and news relating to the project and the wider Duchenne community.

The CARE-NMD team would be grateful for feedback on the new site and would be delighted to work with existing and new partners to develop further translations and localised content in the future. Please contact info@care-nmd.eu if you would like to help!

Updating and disseminating the standards of care for DMD:
ENMC workshop report
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How are care standards for DMD best disseminated, implemented and updated?

What are the bottlenecks for delivery of care recommendations, and what is the best way to overcome them?

These questions were the major topics for the 181st ENMC workshop entitled ”Ongoing updating and dissemination of standards of care for DMD” held in Naarden, the Netherlands, from 10-12 December 2010. The meeting was co-organized by the United Parent Projects Muscular Dystrophy, ENMC, CARE-NMD and TREAT-NMD and included representatives from Western and Eastern Europe and the USA.

The publication of the DMD care recommendations consensus document (The Diagnosis and Management of Duchenne Muscular Dystrophy, Lancet Neurol. 2010 Jan; 9(1):77-93; Lancet Neurol. 2010 Feb; 9(2):177-189) was an important step towards the establishment of a global baseline level of care. Measures must now be taken to ensure their dissemination and implementation. Furthermore, it is crucial that the care recommendations are continuously updated in order to keep pace with updates in best practice so that they retain their value over time.

At the ENMC workshop, an initial overview of  the status of the current DMD guidelines - dissemination, translations, planning for further development - was followed by a session on the roles of CARE-NMD (an EU project to implement best-practice standards of care for Duchenne muscular dystrophy across Europe), TREAT-NMD training courses and education activities, the TREAT-NMD clinical trials coordination centre, MD-Starnet and related US activities, and the French DMD network to help disseminate care standards.

Interesting discussions also arose from presentations on what can be learned from a care perspective from natural history (CINRG, North Star, UPD) and therapeutic studies (Ataluren, cardioprotection, and others), and how tools such as patient registries and the TREAT-NMD Care and Trial Sites Registry can be further developed to act as a resource. In particular, the potential use of “quality of care indicators” assessing key outcome indicators was presented and discussed as a means to make use of the TREAT-NMD clinical trials coordination centre for assessing care implementation.

Similarly, criteria for DMD reference centres were discussed, based on EU expectations for expert centres for rare diseases. The final sessions at the workshop dealt with care areas lacking in the existing document or in need of updating, and how to promote excellence of care and high quality research. It was concluded that the major involvement of patient representatives is crucial for the updating process, and that certain key areas need to be included here, including transition to adulthood, endocrine issues, and oral care. It was also suggested that efforts be made to integrate work on outcome measures to be used for clinical trials with care assessments used in regular clinical settings. A web-based program was presented that would allow for this and that would aid in the process of updating standards of care.

For further information about the process of updating the DMD care standards, contact Thomas Sejersen.

Planning a meeting?
Visit our meetings and events section to avoid date clashes
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The TREAT-NMD website hosts what is now a fairly comprehensive listing of meetings and events worldwide relevant to the neuromuscular field. With both a chronological listing and a monthly calendar view, this listing is a useful resource not only for those wishing to find out details of a particular meeting but also for meeting planning, helping to avoid date clashes between important meetings.

We need your help to ensure this listing is as comprehensive as possible. You can submit an event to the site by filling in our online form, and we do encourage everyone involved in planning a relevant event to mark its place in our calendar as soon as you have fixed your dates.

Awakening Australia to rare diseases: national symposium
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On the 18th-20th of April 2011, Western Australia will host a rare disease symposium, entitled Awakening Australia to Rare Diseases: Global perspectives on establishing a coordinated approach to a national plan. This symposium builds on work initiated by the Australian Paediatric Surveillance Unit (APSU) and is an important step in the process of developing a rare disease strategy in Australia.
The symposium will draw together clinicians, researchers, government policy makers and regulators, industry and disease support and advocacy groups and builds on the work started by APSU and the National Rare Disease Taskforce.
The Office of Population Health Genomics has a mandate from the Director General of Health WA to coordinate a national symposium and begin the process of developing a rare disease strategy for Western Australia. Our motivation for the symposium is to provide information and a framework for a national plan, to use the opportunity for engagement and to build long term relationships with industry, policy, advocacy and support groups, clinical and medical services across the spectrum of rare disease stakeholders. It is only through engagement and developing a strong national voice for rare diseases that we can implement a plan for Australia.

Further details can be found the Rare Disease Australia website.

Optimal Role of Patient Organisations in Drug Development
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Kick-off meeting 24 March 2011

The position of patients has changed from ‘sufferers’ and users of care to partners in care, research and drug development. Over the last decade patient organisations have not only played a role in lobbying but also shown they are willing to shoulder responsibility and contribute towards advancing treatments and a cure. 

Patient organisations have set up patient registries, biobanks and databases with patient-registered outcomes. They have advised on standards of care, trial design and outcome measures. Industry could benefit from collaboration with patient organisations at an early stage in drug development.

This kick-off meeting begins a discussion on how partnership with patient organisations can contribute to more efficient drug development. With over 200 people from industry, academia, policy makers and of course patient organisations expected to attend, early registration for this meeting is strongly recommended via the dedicated website.

Upcoming meetings
Past newsletters
TREAT-NMD conference 2011
28th January 2011
TREAT-NMD newsletter no. 93
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