TREAT-NMD 2016 Review

Dear Readers,

As is customary at the end of the year, we look back at our achievements. Once again TREAT-NMD has had a successful year with many highlights.  We have now implemented our 3 year work plan which is overseen by the Executive Committee We are making fantastic progress in some disease areas, however we need to improve our efforts in other areas. Some tasks are overarching for all NMDs, whilst others are disease specific and sometimes there is coordinated work ongoing in one disease that could help facilitate similar efforts in other diseases. It’s not too late to come forward and volunteer to help- for information contact Becca Leary.

Our website continues to be one of our major portals of communication and in September we welcomed our 1000th twitter follower! In the last year, the website attracted almost 270,000 views from over 67,000 different visitors. Notable shift in site traffic came from Taiwan with 1,248 page hits this year generating more than a 200% increase on the previous. Most traffic came from the USA, European member states, India, Canada, Australia and Japan. The current version of our website is over 6 years old and we have started developing a new website. Given the size and complexity of our current site we’re sure you can appreciate this is no small task that can be undertaken lightly. We feel now is the right time to create something that will not only be more appropriate for today’s technology, but will allow us to work more efficiently and effective in the years to come. Inevitably there will be a number of changes and a period of adjustment as we transition from one site to the other. Please look out for announcements in our newsletters, as we will keep you informed of our progress.

We have steadily increased the variety and number of languages that our information is available in.  The Duchenne Muscular Dystrophy (DMD) family guide is available in 34 languages on the website, the latest version being in Arabic and the online guide is available in 15 languages with Hebrew and Chinese the latest additions. The Spinal Muscular Atrophy (SMA) care guide for families and their doctors is now available in 19 languages with Slovenian being the latest translation. DMD Research Overview contains a succinct overview of therapeutic approaches that are in clinical development for DMD, written in everyday language and are now also available in Dutch and Danish.

During 2016, TREAT-NMD has been able to provide a broad range of support to Pharmaceutical companies, to assist them with post marketing surveillance activities, including strategic and operation input, development of training workshops and communication strategies. To find out more about our complete post marketing surveillance service and our current work on the development of a Duchenne Muscular Dystrophy-specific post marketing surveillance platform, please contact Anne Oyewole

Four TREAT-NMD resources (TREAT-NMD Advisory Committee for Theraputics (TACT) and The Care and Trial Sites Registry (CTSR), Standard Operating Procedures for preclinical efficacy studies and TREAT-NMD Patient Registries achieved IRDiRC recommended status during 2016. TREAT-NMD organized and facilitated a number of meetings and training events, such as the DMD masterclass and the TGDOC members and curators meeting. SMA Europe, TREAT-NMD, and the European Medicines Agency (EMA) co-organised a successful one-day workshop on SMA. The workshop brought together key stakeholders (patient representatives, academics, industry and regulators) to discuss, help and advance the development of therapies for the treatment of SMA.

Looking forward, 2017 will represent a significant milestone; 10 years of TREAT-NMD! We will be celebrating the past and exploring the future of TREAT-NMD at our conference next year. Please join us in Freiburg on the 27th-29th November.I will also take this opportunity to thank the previous chair Annemieke for her contribution during 2016 as well as the rest of the Executive Committee members. I would also like to thank the Secretariat in Newcastle for their work in supporting the Executive, TACT and TGDOC committees. 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 still to be done and we need your help to prepare the neuromuscular field for the future.

Wishing you all a wonderful Christmas and a Happy New Year.

On behalf of Kevin Flanigan


12 Apr 2017