Dear Members and Friends of TREAT-NMD,
As 2019 draws to a close, I, as the new Chair of the TREAT-NMD Executive Committee, would like to take this opportunity to thank you all for your contribution to the TREAT-NMD network. It is an honour and privilege to work with you all.
In the last year major achievements have been made for the network and the neuromuscular field.
First to mention, of course, is the 6th international TREAT-NMD conference, which took place in Leiden this December and was yet another successful networking event. Over 300 representatives from academia, industry and patient organisations attended. As before, the conference had an interactive format to allow all participants to discuss current challenges and align efforts to jointly address and overcome.
Secondly, the TREAT-NMD Enterprise has now been established, giving us independence and allowing us to act as a legal entity in our interactions with companies, regulators and enabling us to be a formal partner in project applications. Volker Straub, Fabiola Bertinotti and Annemieke Aartsma-Rus have been installed as Directors of the Enterprise. Ben Watling has been appointed as the Chief Executive Officer of the Enterprise, and will work under the guidance of the Executive Committee.
The TREAT-NMD website has always been a very useful source of information and this year we launched the new website. Throughout 2020, the TREAT-NMD Secretariat and our wider partners will begin a process of updating the existing information and ensuring the website remains a useful and dynamic resource. If you have any suggestions on how our website could be improved please contact us.
Our membership continues to increase, with currently 829 members, 665 individual and 164 organisations. If you are not yet a member, please take a minute to sign up. Membership is free of charge. Individual members also have the opportunity to nominate themselves or another member to fill a vacancy on the TREAT-NMD executive committee, to vote on prospective committee candidates and vote on the location of future conferences.
The TREAT-NMD Education Committee, which was established in 2018, is running well; 2019 saw two successful TREAT-NMD masterclasses for DMD (Amsterdam, Netherlands) and SMA (London, UK) respectively. For 2020, masterclasses are planned in Sao Paolo, Brazil (DMD) and LGMD (location to be announced). In addition, the 2nd successful TREAT-NMD/EURO-NMD Translational Summer School was held in Leiden, the Netherlands. As before, the Summer School focused on all aspects and stakeholders in therapy development, putting the patient centrally. Another Summer School is planned for July 6-10 2020 (Leiden, the Netherlands).
TREAT-NMD have organised and supported a number of successful meetings throughout 2019 including a meeting of the TGDOC Myotonic Dystrophy Working Group during the IDMC-12 conference in Sweden, DMD Dataset workshops, a TGDOC Outreach meeting at WMS and the annual face-to-face meetings of the Executive Committee and TGDOC.
The TGDOC registries network has had an eventful year, with the development of disease-specific working groups, each Chaired by a relevant disease expert and including a patient representative. These groups will increase the networking activities of TGDOC and will work on disease-specific projects. A significant increase in the number of Global Registry Enquiries has been observed, with a number of further enquiries in the pipeline for SMA, DM1 and FSHD as well as DMD, and are expecting this trajectory to continue into 2020. We would like to take this opportunity to express our heartfelt thanks to all TGDOC Registry Curators for participating in these activities.
2019 was a significant year for TACT as it celebrated its 10th anniversary. A paper was published in the November issue of Nature Reviews and Drug Discovery highlighting the achievements of TACT during the 10 years since its creation. The committee also successfully met twice in 2019, once in Glasgow in April and again in November in Toronto.
TREAT-NMD are developing a Universal Registries Platform. This will be a web-based platform with disease specific modules, which we intend to:
- offer to affiliated registries in our network to use if they wish, to independently host their registry and collect and store their data.
- use to improve the central data collection and analysis process for our global registry enquiries.
Due to reallocation of priorities from partnering pharma companies, development of the Platform has been delayed. TREAT-NMD are seeking additional funding to complete the development and remain committed to completing the Platform before the end of 2020, which will support multiple neuromuscular disease modules.
Many TREAT-NMD colleagues around the world have dedicated time and effort to this work so far, and I would like to thank you all for your contributions, commitment and your continued patience.
I would like to take this opportunity to also thank the past and present members of the Executive Committee for their time and dedication to TREAT-NMD, particularly Janbernd Kirschner, the former TREAT-NMD Executive Committee Chair, I thank him for his time and dedication as Chair. I would like to extend a special thanks to the TREAT-NMD Secretariat in Newcastle for their hard work in supporting the Executive Committee, TACT and TEC, and TGDOC.
Finally, I would like to thank all of you for your continued support of TREAT-NMD. I look forward to working with you in 2020 to help prepare the neuromuscular field for the current therapeutic developments. For now, I wish you all a blessed Christmas and a Happy New Year.
