31st July 2015
Call for nominations to join the TREAT-NMD Executive Committee

In accordance with the TREAT-NMD Alliance charter, the TREAT-NMD Alliance Executive Committee is seeking three additional members; two representing patient organisations and one academic member. If you are interested in nominating yourself or a colleague please send your nominations to Stephen Lynn by Wednesday 16 September 2015 and provide a short supporting statement. 

Nominees are expected to be or willing to become members of the TREAT-NMD Alliance and agree with the TREAT-NMD Alliance Charter. All TREAT-NMD Alliance members (as of September 16) will be invited to vote for their preferred candidate. The successful candidates will be announced in October and join the TREAT-NMD Alliance Executive Committee to help with the strategic planning and direction of the Alliance. The new Executive Committee will meet in person immediately after the TREAT-NMD Conference on 8 December 2015 in Washington DC.

Please consider taking on this vital role and becoming a member of the TREAT-NMD Alliance’s Executive Committee.

If you have any questions about the Executive Committee please contact the Secretariat at Stephen Lynn.

Revised Duchenne guidelines for anaesthesia and vaccines
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Parent Project Muscular Dystrophy have been working to revise anaesthesia and vaccine guidelines and have now published their recommendations. New evidence has suggested that while rhabdomyolysis (breakdown of skeletal muscle tissue causing the release of myoglobin, a muscle protein that damages the kidneys) may still be possible with inhaled anaesthetics, it is rare, and unrelated to malignant hyperthermia (“MH”; extremely high body temperature).

Find out more information about the new recommendations for anaesthesia.

The Centers for Disease Control and Prevention in the United States have very recently updated their recommendations for vaccinations for people living with Duchenne. The new recommendations should be considered carefully and aim to help to answer the regularly asked questions, “to give or not to give, when to give, how to give and what to give.”

Comprehensive information can be found on the PPMD website.

TREAT-NMD Conference 2015. Book your space now!
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Growing the drug pipeline for neuromuscular diseases: optimising resources for clinical development of new therapies - 6th-8th December 2015

The TREAT-NMD International Conference is again designed to address some of the key challenges facing the neuromuscular community as we move forward with developing new therapies for patients. Each session will be highly interactive with the audience, who will represent all key stakeholder groups: patient organisations, academics, clinicians, industry and regulators. The sessions will briefly address and introduce the state of the art in each area and then interactive discussions with the session participants and audience will address key topics facing the community. Topics include; the challenges of trial design, outcome measures, standards of care, post-marketing and registries. As in previous conferences, these discussion sessions ensure there is maximum interaction between the audience and the invited experts.

The Conference will be held at the historic Cosmos Club in the heart of the DuPont Circle region of Washington DC. Participation is strictly limited to 200 with tickets selling fast. Be sure to avoid disappointment and secure your space by booking now.

The conference hashtag is now live #TNMD2015 follow us to keep up to date with all the conference news.

European Comission seeks to support member states
in the development of equal access health networks
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There have been many efforts at a European level to improve the profile of rare diseases (RD) and to develop mechanisms which will allow care for patients with RD to be better organised. One of these initiatives is the creation of European Reference Networks (ERNs). ERNs have been mandated in the Directive on Patients' Rights in Cross-Border Healthcare in 2011. The main aim of an ERN is to promote equal access to the most up-to-date care and advice to all individuals, regardless of where they live in Europe.

In the neuromuscular field much has already been done regarding networking; however, most of the work was done on a research basis and it is now time for the field to join efforts and organize ourselves in a ERN both for care and research.

The European Commission is seeking to support EU Member States in the development of ERNs and is presently putting together a series of initiatives which include the 2nd European Reference Networks Conference: from planning to implementation that will be held on the 8-9 October 2015 in Lisbon with the first call for networks expected in early 2016.

Find out more about the timeline involved and possible opportunities for the neuromuscular field.

Highlight on upcoming SMA Awareness activities
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August is the Spinal Muscular Atrophy (SMA) Awareness Month in the US. This year CURE SMA , the biggest SMA organisation, has planned a number of events throughout the month, including the SMA candle lighting on 8 August. To help those wishing to participate and raise awareness they have also developed a series of tools which can be downloaded from their website, these include the Awareness & Advocacy Kit and the Medical Professional Awareness Kit. Cure SMA will also be sharing facts, information, and ways to get involved on their website and on social media throughout the month.

On the other side of the Atlantic, awareness raising activities in the UK will taking place between 28 September and 4 October. This has been scheduled to tie in with the SMA related activities in other European countries. Find out more about what's planned in the UK by visiting the SMA Support UK and SMA Trust websites.

Detailed information about the activities planned for the SMA Awareness Month in August in the USA can be found on the Cure SMA website here.

TREAT-NMD Advisory Committee for Therapeutics
(TACT) call for proposals
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The 12th TACT review meeting will take place between 5-6 December 2015 in Washington DC, forming part of the larger TREAT-NMD conference (6-8 December).

The deadline for submitting full applications for TACT has been extended to the 25 September 2015.

Those wishing to submit a proposal or know of someone who would benefit from submitting a proposal to this meeting should contact the TACT secretariat via Kim Down as soon as possible. Please visit the TACT section of our website for more details of the application process.

During this review meeting Dr Kathryn Wagner will take over as Chair of TACT from Professor Dominic Wells as he concludes his two year term. The secretariat, core and extended membership of TACT as well as TREAT-NMD as a whole would like to take this opportunity to thank Professor Wells for his dedication and excellent work at this time.

Landmark strategy meeting aims to boost UK trial capacity
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The UK National Workshop on Duchenne Muscular Dystrophy Clinical Trial Capacity was held at Newcastle University on the 10 July 2015. Representatives from patient organisations, pharmaceutical companies, the National Institute for Health Research and a wide range of clinicians met to discuss the challenges and solutions to increase clinical trial capacity in the UK.

The outcome was the 'Newcastle Plan' which outlines three phases of development to be taken forward by a working group derived from the meeting participants.

The five year objective of the plan is to ensure that all patients with DMD, both children and adults, have access to clinical research opportunities. Read the summary document from the meeting.

Applications for scientific officer post being taken
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Applications are now being taken for the position of scientific officer at the John Walton Muscular Dystrophy Research Centre based in Newcastle, UK.

Directed by Prof Hanns Lochmüller, Prof Kate Bushby and Prof Volker Straub the successful applicant will join a multidisciplinary team to develop and lead on translational research in neuromuscular disorders, and will be expected to contribute significantly to the scientific output of the centre through their own research into inherited, neuromuscular disorders.

A scientific background (e.g. PhD), experience in –omics research, gene and cell therapy, and insight on how new developments elsewhere impact on the work of the centre will be crucial.

A proven track record of effective communication and presentation skills, scientific supervisory experience, grant and publication record are also very important.

Informal enquiries should be directed to Hanns Lochmüller

Application closing date - 6 August 2015

FSHD Global to host scientific information evenings in Australia
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The FSHD Global Research Foundation is hosting a number of scientific information evenings across Australia in September. These events are an opportunity for patients to learn about recent research developments and progress into clinical trials and treatments, as well as allowing families and patients to share knowledge and insight. Updates will be given by the world leading FSHD clinicians and researchers. This will include members of the TREAT-NMD task force, Baziel van Engelen, Stephen Tapscott, Rabi Tawil, Sabrina Sacconi and current TACT chair Kathryn Wagner among others.

The information evenings will take place in Sydney on 22 September, Melbourne on 23 September and Gold coast on 24 September. For more information and to register visit the FSHD Global website.

Limited places are still available for DMD expert masterclass
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3-4 September - Amsterdam

The progressive and debilitating nature of Duchenne muscular dystrophy (DMD) means that timely diagnosis and effective care are integral to improving the prognosis and quality of life of patients with this life-limiting condition. This 2-day expert masterclass will provide a high-quality scientific meeting for paediatric neurologists who may encounter patients with DMD.

The expert masterclass will consist of presentations by experts in the field and interactive workshops, and will fulfill the following objectives:

- To underline the importance of full mutation-specific diagnosis of DMD
- To review current understanding of the natural history of DMD and best practice for the management of patients at various stages of ambulation and non-ambulation
- To discuss methodologies for the evaluation of treatment outcomes
- To update on therapeutic advances in DMD and potential implications for clinical practice

Register now to avoid disappointment!

For further details and to register, please contact Sally Janani

Telephone: +44 203 675 7073

TREAT-NMD have received an unrestricted educational grant from PTC Therapeutics International Ltd for the organisation of this meeting

TV star uses own disability experiences to write children's book
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The star of one of the most loved Children's TV shows in the UK is using her own experiences of disability to help children understand what it’s like to be different and how to embrace their own uniqueness.

Kim Tserkezie who played Penny Pocket in the BBC's Balamory has written her first children’s book. “Toys for Tomorrow” the first book in the series, is about young Millie, a wheelchair user who has a love for adventure and an amazing Grandad who helps her see how, with a little guidance, even the cheekiest little ones can channel their energy into something positive and wonderful. Kim is now taking the time to talk to children about the book and her experiences of disability and how she became successful in her chosen career.

Find out more about the book from the publisher's 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. Don't forget you can always find out what we're up to by following us on Twitter.

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.

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Past newsletters
31st July 2015
TREAT-NMD newsletter - 31st July 2015