TREAT-NMD 2015 review

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 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,

Annemieke

 
12 Apr 2017