17th December 2015
Message from Annemieke our TREAT-NMD Chair

Dear Readers,

As is customary at the end of the year, we look back at our achievements. I am happy and grateful that 2015 was again a year in which a lot of progress has been made for care, research and therapy development for Neuromuscular Disorders.

Our most recent highlight was of course the successful, sold out TREAT-NMD International Conference that took place in December in Washington DC (read more about this below). A personal highlight was the Scientific Award that was given to one of the founding TREAT-NMD Coordinators, Professor Kate Bushby by EURORDIS during the EURORDIS Awards in Brussels.

However, there were other highlights as well, e.g. the fact that 10,000 virtual balloons were sold to raise DMD awareness on the 2nd Global Duchenne Awareness Day (Sept 7), and other awareness efforts such as the first annual LGMD awareness day (Sept 30) and the SMA awareness month (August). TREAT-NMD published two significant papers on TACT and the utilization of the TREAT-NMD Global DMD Registries which highlighted unequal access to best-practice care for adults with DMD in 7 European countries.

Furthermore, our tools continue to be valuable as outlined by their use: TACT reviewed 11 applications, including potential therapies for DMD, SMA and mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). Our website was visited more than ever, with 285,000 page hits last year generated by 68,000 different people from 185 different countries. Areas that have shown significant increase in include pages on clinical trial information in various diseases, namely DMD, SMA and LGMD. Our social media presence continues to gather strength and we expect to have 800 Twitter followers in the New Year.

TREAT-NMD organized and facilitated a number of meetings and training events, such as the first DMD masterclass and an international clinical outcome study for dysferlinopathy. Our collaboration with regulatory agencies was continued through a stakeholder meeting on exon skipping hosted by EMA, and TREAT-NMD initiated work on dystrophin quantification was presented at an FDA workshop on dystrophin. Also, webinars to discuss post marketing surveillance and to prepare for the first call for European Reference Networks (ERNs) were hosted by the John Walton Muscular Dystrophy Research Center.

While TREAT-NMD started as a European project, with a primary focus on DMD and SMA, we are now more global and branching out to more diseases, as underlined by our involvement in disseminating the American Academy of Neurology (AAN) and the American Association of Neuromuscular & Electrodiagnostic Medicine's (AANEM) guidelines on FSHD. We also promoted the new practice brief focusing on foot care for people with CMT and promoted a new study to identify people living with genetic disorders in New Zealand (including GNE myopathy, DMD, BMD and LGMD). For the future, we intend to continue these efforts.

After looking back, one cannot help to look to the future. For 2016 the first order of business will be to update the workplan: we will maintain useful tools, and hope to develop them for additional neuromuscular disorders. As you know TREAT-NMD is a collaborative network of all stakeholders, researchers, clinicians and patient representatives and I sincerely hope that I can count on all of you to help us with these efforts. Now that many drugs are tested in clinical trials, we are also preparing for a time where medicines are available for neuromuscular disorders. Our work on generating disease specific post marketing surveillance databases will continue in collaboration with CINRG. Furthermore, we will focus on compiling natural history data that is available in the community. If you are willing and able to help out with our work, please contact me or the secretariat. We are all part of TREAT-NMD!

I will also take this opportunity to thank the fantastic team working at the Secretariat, who make chairing the network easy and enjoyable. I want to thank Kevin Flannigan who will take over as Chair in October 2016. Furthermore, I want to thank Kathy North, Anna Kole and Alejandra Pereda for their efforts as EC members and want to welcome our new EC members: Nathalie Goemans, Petra Palmgren and Yuriko Oda. I look forward to working with you and to continue working with other members of the executive committee. Your time and effort is much appreciated.

Finally, I also want to thank all of you for being part of TREAT-NMD and want to stress that you can be involved in many ways. There is a lot of work to be done and we need your help to prepare the neuromuscular field for the future.

Wishing you all blessed Christmas and a productive 2016,


TREAT-NMD International Conference 2015 Report
back to top    

The TREAT-NMD International Conference was held in Washington DC from 6-8 December 2015 with the theme of growing the drug pipeline for neuromuscular diseases: optimizing resources for clinical development of new therapies.

As in previous conferences the agenda was designed to address some of the key challenges facing the neuromuscular community as we move forward with developing new therapies for patients. Each session was highly interactive with the audience, who represented all key stakeholder groups: patient organizations, academics, clinicians, industry and regulators.

The sessions addressed the following:
- The Essential Resource in Achieving Novel Therapies for Neuromuscular Disease: Patient-Focused Drug Development
- The current challenges of preclinical research and study design
- Innovative trial designs and outcome measures
- Biochemical and imaging outcomes
- Early approval and Post-marketing: Rethinking the registries in orphan disease
- Standards of care
- Registries
- International trial readiness and access to emerging therapies

The conference was attended by over 220 delegates and this was the first TREAT-NMD Conference to be held outside of Europe. We would like to thank all the speakers and the session chairs for delivering an excellent programme, which you can find on the TREAT-NMD Conference web site.

Alongside the TREAT-NMD Conference we also held meetings of the TREAT-NMD Global Database Oversight Committee (TGDOC), TREAT-NMD Advisory Committee for Therapeutics (TACT) and the TREAT-NMD Executive Committee. Thank you to all who attended these meetings in Washington DC and who helped make the meetings a huge success!

Second MYO-MRI Training School
back to top    

The 2nd MYO-MRI Training School which will be held 15-17 June 2016 at the Institut de Myologie in Paris is now open for registration.

The MYO-MRI project, "Applications of MR imaging and spectroscopy techniques in neuromuscular disease: collaboration on outcome measures and pattern recognition for diagnostics and therapy development", is led by Volker Straub and Pierre Carlier and funded by COST, the European Cooperation for Science and Technology. It aims to overcome the main hurdles to rollout of MR techniques by sharing expertise and data, validating protocols across platforms and exploring the potential of MRI and MRS as a helpful diagnostic tool and a quantitative outcome measure in NMD clinical trials.

The training of early stage researchers is an important aspect of the MYO-MRI project and we are pleased to announce that the 2nd MYO-MRI Training School is now open for expressions of interest.

The three-day course will give an in-depth introduction into the four Working Groups that make up the project on day one, with days two and three focusing on a range of Working Group specific modules, taught by experts from the MYO-MRI project itself.

A programme of the specific modules is currently being finalised.

The Training School is now open for expressions of interest, and we encourage Early Stage Researchers in particular to apply.

If you are interested in attending, you can register your interest here.

Expert Patient and Researcher EURORDIS
Summer School 2016 - Applications open
back to top    

Applications are now open for this year's EURORDIS Summer School held from 6-10 June in Barcelona.

Since EURORDIS launched its training programme committed to empowering those living with rare diseases in 2008, the Summer School has trained over 300 participants from over 40 countries and representing more than 70 diseases. "ExPRESS 2016" will bring researchers and expert patient representatives together to discuss and learn about the development of medicines. The 4 and a half day capacity-building programme is sure to be an exciting and insightful experience, covering clinical research and access to orphan, paediatric, advanced therapies and health technology assessment.

For more information on this fantastic opportunity and how to apply please visit the EURORDIS website.

The deadline for applications is the 31 December 2015.

MEET Symposium - fellowships to attend available for short time
back to top    

The MEET Symposium 2016 'From bench to bedside, and back' will see patients MEET Researchers.

The Mitochondrial European Educational Training Project (MEET) is funded within the frame of PEOPLE Marie Curie Actions. The symposium will be held at Radbound University Medical Centre in The Netherlands. Click here for additional info and here for registration.

MEET consortium offers 15 full fellowships to attend; covering travel, accommodation and subsistence costs to attend the Symposium. Fellowships are addressed only to patients or Representatives of Patients’ organizations.

Applicants must submit their requests together with a reference letter from the patients’ organization with whom they are collaborating. Applications should be sent to Serena Paterlini.

The application deadline is December 20, 2015 at 18.00 (Brussels time).

Myology 2016 - early bird registration open
back to top    

Early bird registration for the Myology 2016 congress closes on 31 January 2016!

The best specialists from around the world will gather in Lyon, France to present and challenge their latest findings not only in fundamental research but also in clinical science and therapeutics.

Further details about the programme, along with important dates to remember, are available on the Myology 2016 website.

You are encouraged to register as soons as possible to ensure you take full advantage of the early bird discounted rates which are only available for a short time.

Opportunity for clinical scientist with experience in muscle pathology
back to top    

An exciting opportunity has arisen for a highly motivated scientist to provide a specialist technical service for the diagnosis of Limb-Girdle Muscular Dystrophies (LGMDs). The Muscle Immunoanalysis Unit (MIU) is the UK's national referral centre for the diagnosis of LGMDs, where immunohistochemistry and western blotting techniques are used to help identify which gene is at fault. The MIU works closely with the diagnostic molecular genetics unit and clinicians at the Institute of Genetic Medicine, Newcastle upon Tyne in the UK. The unit supports an active R&D programme. Applying candidates should have specialist experience of muscle pathology and associated diagnostic techniques.

UK candidates should be state registered with the Health Professions Council or last year trainees. Equivalent qualifications for overseas candidates can be used to support an application for state registration.

To apply please go to NHS Jobs website for more details. If you would like an information pack or further information, contact Dr Richard Charlton (Clinical Scientist) or telephone 0191 2820849.

Submit an article
Past newsletters
17th December 2015
TREAT-NMD newsletter - 17th December 2015