TREAT-NMD End of Year Statement 2022
Dear Members and Friends of TREAT-NMD,
As we come to the end of 2022 we would like to wish all our members a merry Christmas and best wishes for the new year.
2022 has been another busy year for TREAT-NMD. We’d like to thank the TREAT-NMD Executive Committee for all their continued support and hard work this year. We have now consolidated the not-for-profit status of TREAT-NMD Alliance Ltd by registering with the Charity Commission in the UK. In addition to this we have made the exciting transition to establishing the Board of Trustees for TREAT-NMD Alliance Ltd and forming the NMD Advisory Committee (NMDAC). The NMDAC will develop the strategic neuromuscular disease framework that will in turn support trustee decision making. You can find out more about our activities in our first annual Impact Review
It was great to end the year with our first conference since 2019. The conference was a fantastic event where more than 200 colleagues from across the neuromuscular community came together in Vancouver, Canada for three days of networking, interactive sessions and talks. Highlights included our fantastic programme of speakers, the spectacular waterfront venue in Vancouver and all the opportunities to meet with colleagues and friends in person.
The TREAT-NMD Global Registry Network has continued to grow this year, with 67 registries now being part of the Network. The TGDOC Governing Committee has offered great support to the team this year, and we would particularly like to thank Craig Campbell for all his work as one of the TGDOC Chairs over the past 6 years. The network has completed four registry enquiries this year, but has also started some large scale projects requiring analysis of patient-level data. This work will continue as we support post authorisation studies in the future. We have also built close links with the EMA, as we work towards qualification for Global SMA Registry Network. In November we received a letter of support for this work, and we are looking forward to taking the next steps in 2023.
Back in 2021 we launched the Global Registries Platform and we are pleased to say that there are now 9 registries using the platform to collect patient data for one or more of our expanded core datasets. Other registries have been working towards adopting our expanded core datasets, 17 registries are collecting the expanded core DMD dataset, 10 are collecting the expanded core LGMD dataset and 25 registries are collecting the expanded core SMA dataset.
2022 also saw our first in person TREAT-NMD Advisory committee for Therapeutics (TACT) meeting since before the pandemic. The panel met in Bari, Italy back in April where expert reviewers provided a platform for advice and discussions for the applicants. Alongside the April meeting we also ran a series of virtual TACT meetings in November, the combination of both online and in-person meetings gives us more opportunities to be flexible as well as being able to maintain the important social aspects of being able to meet in person. We would like to thank the TACT core committee and all the additional reviewers that took part. Next year we are hosting an in-person meeting from April 21st-23rd in Philadelphia, USA as well as four virtual meetings in the Autumn.
2022 was a great year for successful TREAT-NMD education programmes. We have launched the TREAT-NMD e-learning platform; a free platform aimed at educating healthcare professionals involved in the diagnosis and treatment of patients with neuromuscular disease.
We also organised several successful expert masterclasses in different disease areas across the year: In February we ran a congenital myopathies Masterclass where over 100 delegates joined a series of interesting talks and interactive workshops. In March we hosted a Duchenne Muscular Dystrophy Early Diagnosis Seminar for a European audience. May was the Limb Girdle Muscular Dystrophy Australasia Masterclass, this 2-day event was chaired by Professor Ichizo Nishino from the National Centre of Neurology and Psychiatry, Japan and Dr Gina Ravenscroft from the University of Western Australia, Australia and by featuring multiple live translations in Mandarin Chinese, Japanese, Korean and English we managed to be inclusive to a new, wider audience of international delegates.
Other good news this year is that TREAT-NMD membership numbers continue to increase, with 1084 members; 890 individuals and 194 organisations at present. If you are not yet a member, please take a minute to sign up. Membership is free of charge and individuals can nominate themselves or a colleague. Members can fill a vacancy on the NMD Advisory Committee, vote on prospective committee candidates, and vote on the location of future conferences.
In terms of projects, over the past year the TREAT-NMD project team has been very excited to collaborate with various partners including regulatory authorities, pharma industries, CROs (contract research organizations), patient advocacies and others. Using the TREAT-NMD’s datasets, we were able to participate in PMS (post marketing surveillance) projects, and new discussions are on-going for projects to start in 2023. Additionally, the team has been working closely with patient organizations to identify collaborative ways to de-risk investments in innovations, to overcome challenges of running educational events and to align datasets globally. With support from key opinion leaders, we are also in process of reviewing standard operating procedures (SOPs). The SOPs are being developed to help with preclinical research and to avoid duplication of effort and to share best practice.
We would also like to welcome the new team members that have joined the growing team at TREAT-NMD Services Ltd over the past year. As we continue to expand, we can look forward to new projects, events and collaborations into the next year.
Looking forward to 2023 we have some exciting events and projects in the pipeline. The limb girdle muscular dystrophy virtual masterclass will take place on March 9th and 10th 2023 and is aimed at a North American Audience. Plans are also underway for a 5 day in person masterclass taking place from the 22nd to the 26th of May in Amsterdam. Across the week, experts will cover Duchenne muscular dystrophy, gene therapy and spinal muscular atrophy. Delegates can pick and choose whether they will attend one, two or all three topics.
In summer we are planning our first masterclass in FSHD and later in the year a Pompe masterclass for a Latin American audience. The best way to keep up to date with all TREAT-NMD events is to follow us on Twitter and LinkedIn where you will see all the latest updates.
Before concluding, we would like to acknowledge all in the neuromuscular community who passed away in 2022, and to reaffirm our pledge to honour these memories by continuing to work to find and advance treatments and improve care for individuals living with neuromuscular disease. Finally, thanks to everyone whose hard work contributed to 2022 such a successful year for TREAT-NMD. Wishing all fantastic holiday and a Happy New Year!
Jim Dowling, Chair of the TREAT-NMD Executive Committee
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David Allison Chief Executive of TREAT-NMD