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29th November 2013
TREAT-NMD Alliance Conference - a report

Last month’s TREAT-NMD Alliance Conference in Newcastle brought together approximately 200 delegates from over 30 countries. Attendees consisted of patient representatives, academics, clinicians, patient registry curators, industry representatives and other key experts, all with an interest in neuromuscular disease. We would like to thank all the speakers and participants for their invaluable contributions all of which made the meeting a great success.

The conference was divided into three sessions. The first of these focused on the results and achievements of TREAT-NMD related research and included CARE-NMD results, Burden of Illness in DMD, impact of TACT, AON clinical trials in DMD, new outcome measures for SMA and DMD, natural history studies and therapy development in GNE myopathy, as well as a presentation about what has been learnt from DMD registries.

The second session was devoted to patient involvement and ethics, and included a preview of the forthcoming patient forum followed by a presentation entitled: ‘Advocacy, expectations and hype: improving the communication of clinical results in neuromuscular diseases’.

The third session looked at the upcoming new themes of collaborative research, covering topics such as next generation sequencing, emerging therapies for a number of diseases, MRI as an outcome measure, promising biomarkers and the use of patient registries. All sessions generated very interesting and in-depth discussions.

For those who could not attend the event, the full programme of the conference along with some of the presentations are now available on the TREAT-NMD website.

The conference was not only an opportunity to learn about the past, current and upcoming themes of collaborative research in the field but it also provided a unique environment for networking and discussions. The feedback received so far has indicated that having an opportunity to network during the event was very much appreciated by delegates.

We would like to thank all those who completed the conference feedback form - we value your opinions. If you attended the conference but haven’t yet provided us with your comments, please get in touch with Agata Robertson for a link to the anonymous on-line feedback form.

New Chair of Executive Committee - Annemieke Aartsma
Vice Chair - Eric Hoffman
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We would like to officially announce that Annemieke Aartsma-Rus has now taken over from Hanns Lochmüller as the Chair of the TREAT-NMD Alliance Executive Committee with Eric Hoffman now the new Vice-Chair. Annemieke and Eric formally adopted their new roles at the face-to-face Executive Committee meeting which was held on 1 November 2013 in Newcastle, UK.

We would like to take this opportunity thank Hanns for his excellent work as the first Alliance Chair and his role in shaping the TREAT-NMD Alliance during this time. Since January 2012, the network has developed into a fully fledged Alliance, with a governance structure consisting of an elected 13-member Executive Committee, a new charter and a highly successful new membership scheme

Along with the Executive Committee members, a number of Taskforce members also attended this very productive meeting. The main focus of the meeting was on our three year action plan which now has only one year left in its current form. With this in mind, work has already begun on updating the action plan, which once ready will be published on the TREAT-NMD website.

Annemieke commented that ‘“these are exciting times, we are learning a lot from ongoing and completed trials and we continue to be committed to move the neuromuscular field forward”.

TREAT-NMD Alliance Executive Committee
voting to begin to fill two vacancies
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Voting begins next week to elect two new members of the TREAT-NMD Alliance of the Executive Committee. The recent retirement of Hanns Lochmüller and Anna Ambrosini from the Executive Committee has opened up two vacancies: one for an academic representative, and one for a patient organisation representative.

On behalf of the TREAT-NMD Alliance, we want to thank both Hanns and Anna for their work, and to especially thank Hanns for chairing the Executive Committee since January 2012. Both Hanns and Anna will continue to work with and support the activities of the TREAT-NMD Alliance, and we look forward to welcoming their replacements to the Executive Committee before Christmas.

We received an excellent response to our call for nominations: the full list of nominees is now available. Voting will be open for approximately two weeks, and is open to all TREAT-NMD Alliance members (as of 1st December 2013), who be contacted next week with information on how to cast their votes.

We will announce the result of the vote in our final newsletter of 2013, which is due to be published on Wednesday 18th December.

Duchenne policy forum
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Earlier this year, the EMA held a public consultation on a draft guideline for clinical investigations of medicinal products for the treatment of DMD/BMD. The TREAT-NMD Alliance organised a workshop in London in June 2013, to bring together stakeholders and EMA committee experts to discuss issues such as efficacy measurements and strategies and design of clinical trials.

Parent Project Muscular Dystrophy (PPMD) is hosting a Duchenne Policy Forum on the 12th December 2013 in Silver Spring, MD. “Optimising Clinical Trials in Duchenne Muscular Dystrophy in the New Era of Improved Care Standards” is designed to create draft guidance for the FDA that reflects the combined experience and wisdom of the Duchenne community. Key Duchenne community stakeholders, including the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), and review divisions, are committed to the success of this meeting.

Experts will present across four main areas: Unmet medical needs; Duchenne natural history in relation to clinical trial endpoints; Biomarkers and potential surrogate endpoints; Framing the draft guidance for optimizing Duchenne clinical trials.

New publications on TREAT-NMD SMA Registries
and the Care & Trial Site Registry (CTSR)
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Two recent publications have featured the development and use of two important TREAT-NMD platforms for translational research: the global SMA registry and the Care and Trial Site Registry.  

"Mapping the differences in care for 5,000 Spinal Muscular Atrophy patients, a survey of 24 national registries in North America, Australasia and Europe", published in the Journal of Neurology, desribes the design, setup and utilisation of the TREAT-NMD national patient registries. It analyses a selection of clinical items, providing a snapshot of data by SMA subtype, and compares the results with published care recommendations.

"The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases", published in the Orphanet Journal of Rare Diseases (Open Access), describes the development of the TREAT-NMD Care and Trial Site Registry (CTSR) and its utilisation for academic research and industry enquiries.

These publications follow on from the publication on the national DMD registries featured in the September Newsletter.

All papers can be downloaded via the TREAT-NMD website

Patient registries section updated
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We are delighted to announce that the patient registries section of our website has been significantly updated. The new section has been specifically created to allow users to find a particularly disease registry or relevant registry information such as our recent publications, as easily as possible.

TREAT-NMD, along with many patient organizations, are experienced in the creation, implementation and curation of registries for neuromuscular conditions. To share our experience we have redeveloped the registries toolkit section. This section provides a detailed but easy to follow outline of what things should be considered when setting up a registry and it also includes example documentation.

It is very important that patients who wish to join a registry can find the correct information as easily as possible. With this in mind, we would like to ensure that information about national and international registries is up-to-date. To facilitate this, the registry section allows to the registry curators to update the information about their registry by completing the form available on the website in the registry section. We would encourage all registry curators to have a look at the pages devoted to their registry on the TREAT-NMD website to check if the information is correct.

A report from the first national meeting of the
neuromuscular registry in Sweden
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The first national meeting of the Neuromuscular registry in Sweden was held in Örebro on 14th November. The new, updated version of the registry and related issues were discussed. A separate registry for DMD was initially started, but its development was delayed in order to enable it to cover other neuromuscular diseases. At the meeting, Björn Lindvall, reponsible for the registry and a TREAT-NMD curator, together with Lisa Quensel, responsible for the registry platform, presented the updated current features of the neuromuscular registry that is now up and running in its expanded format. The registry includes SMA, DM1, congenital myopathies, congenital muscular dystrophies, and LGMD, in addition to DMD.

For DMD and SMA, registry items include both TREAT-NMD mandatory and recommended items, along with some additional information. Of the 202 patients already registered, most have DMD or BMD. Symtomatic DMD carriers, LGMD, congenital myopathy and those with an SMA diagnosis also feature. The steering group for the registry, which is based on reporting by professionals, consists of 14 members of which 2 are patient representatives. Other topics on the agenda were reports from a national screening programme of LGMD and Pompe disease (Hasan Bacin), news about FSHD (Christopher Lindberg), a report from TREAT-NMD (Thomas Sejersen), clinical trials for DMD (Mar Tulinius), and reports from DM1 meetings (A-K Krogsmark, A-B Ekström, and C Lindberg).

Poster abstract deadline for ECRD 2014 approaches
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The European Conference on Rare Disease and Orphan Products will take place in Berlin, Germany, between 8-10 May 2014. The deadline for poster abstract submission is 15th December 2013.

ECRD 2014 will allow everyone from patients, to policy makers, healthcare professionals, industry, regulators, researchers and academics the opportunity to meet one another, exchange information and ideas and join together in the fight against rare diseases.

The conference boasts over 100 speakers and along with numerous professionals in attendance. Topics will include latest research, developments in new treatments and information regarding innovations in health care, social care and support at both the European and national levels. Further details can be found on the conference website in the programme section.

Save the date for the TREAT-NMD workshop
Outcome measures in clinical trials in myopathies
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The International Congress of Neuromuscular Diseases (ICNMD) is held every four years in different locations around the world. It is the meeting of Research Group on Neuromuscular Diseases-World Federation of Neurology (RGNMD-WFN) which is now in its 50th year. It is firmly established as a unique opportunity to share scientific advances between those involved in improving care, understanding disease pathogenesis, and developing innovative treatments in muscle, neuromuscular junction, peripheral neuropathies and motor neuron diseases.

The next congress is scheduled for 5-10 July 2014 in Nice, France and is combination of plenary sessions, seminars, teaching courses and workshops.

As part of the meeting, TREAT-NMD is organising a workshop entitled 'Outcome measures in clinical trials in myopathies' which will be held on Thursday 10th July 2014 in the afternoon.

Early bird registration discount will apply until 28th February 2014, once registration opens. Further details about the congress can be found on the dedicated website. More detailed workshop information will be published in future TREAT-NMD newsletters and on our website in due course.

Upcoming meetings
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29th November 2013
TREAT-NMD newsletter - 29th November 2013
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