To All our Members and Friends,
We want to begin with a very heartfelt thank you to everyone who has given their expertise, advice, and very valuable time, to help us achieve our vision and mission to advance the diagnosis, care, and treatment for those living with neuromuscular diseases. We would not exist without you!
2023 has been marked by significant accomplishments that have propelled us closer to our vision and mission. We continue to build on the success of our 7th International Conference in Vancouver, Canada. It was an honour to host the event and to bring everyone together to share and discuss the latest research, science, and standards of care in neuromuscular disease.
The conference was our most successful to date with over 230 delegates from 35 countries arriving at the Vancouver Convention Centre to take part in sessions planned by the Programme Committee who created a wonderful agenda for our event. One delegate said, ‘The collaborative nature of the conference was incredible! You supported the best way to move forward towards more therapeutic developments, by all stakeholders coming together and learning from one another.’ Hearing from Erin Ward (MTM-CNM Family Connection), Terry Pirovolakis (CureSPG50) and Rick Ferguson and Dr Michelle Lorentzos (Daniel Ferguson Foundation) during our first session ‘Patient engagement’ was profound, highlighting the importance of the patient and family voice.
We are now planning the 8th International Conference to be held in Dubai, UAE between 6th – 8th February 2025.
2023 has been a very successful year for education at TREAT-NMD. In March, a virtual LGMD Masterclass was held, chaired by Chris Weihl and Linda Lowes. It featured a range of discussions, lectures and workshops attended by representatives from 31 countries.
In May, the first TREAT-NMD Masterclass Week took place in Amsterdam. This special event featured masterclasses in DMD, Gene Therapy and SMA hosted at the same venue over five days. Each masterclass welcomed over 70 international delegates to learn about the latest developments, emerging therapies, and standards of care in these areas. The week was an excellent opportunity for delegates to share ideas and connect with other healthcare professionals to strengthen the community in which we work.
In early December we were delighted to launch two new initiatives. The first was our new website POD-NMD which is an education resource hub designed for physiotherapists. Created in partnership with Anna Mayhew, POD-NMD hosts a range of interactive e-learning modules which aim to upskill physiotherapists working with neuromuscular patients. This incredible resource is free to access and features guided interactive modules on functional scales, podcasts and much more. The second was the TREAT-NMD Expert Becker Muscular Dystrophy Masterclass in Barcelona, Spain. With over 80 individuals from the neuromuscular community from across 24 countries in attendance the event was a resounding success. We hope to host future masterclasses dedicated to BMD in other regions very soon.
In addition to our expert masterclass programme, the TREAT-NMD e-learning platform continues to grow, with a reach of over 430+ learners across 53 countries. We were delighted that TREAT-NMD was shortlisted as a finalist for a ‘Talent-LMS ‘Positive Impact’ award for our work. Our e-learning platform is a free resource aimed at educating healthcare professionals involved in the diagnosis and treatment of patients with neuromuscular disease. There are now 27 interactive learning modules for clinicians on LGMD, DMD, CM and SMA. Also, a series of modules for physiotherapists with videos and training guides for a variety of functional assessment scales including downloadable resources for use in clinic.
The Education and Events Team are looking forward to 2024 with exciting new projects in the pipeline: we plan to host masterclasses across several regions including events dedicated to FSHD, LGMD, DMD and Gene Therapy and will continue to add modules to our e-learning platform to ensure our education programme benefits everyone.
TREAT-NMD Global Registry Network
Throughout 2023, we have been hard at work on publications and empowering registries to lead research initiatives. This commitment bore fruit with the annual registry review survey, providing invaluable insights that we proudly presented at the prestigious World Muscle Society conference.
The TGDOC Governing Committee has offered great support to the team this year and we would particularly like to welcome Miriam Rodrigues as an incoming TGDOC Chair. Looking ahead, improving data quality remains a cornerstone of our vision for 2024, underlined by the anticipation of exciting new data projects.
We ended the year on a high with our annual registry curators meeting in Amsterdam. Over 45 dedicated curators from our expansive registry network joined us for two days of collaborative discussions, networking, and the invaluable opportunity for disease subgroups to meet in person. This gathering showcased the remarkable growth of our Global Registry Network, now spanning 65 members across 41 countries, with the addition of three new members in the past year alone. The key theme of our meeting revolved around data quality, with TREAT-NMD laying out exciting new quality plans for 2024 and beyond.
Among our ongoing large-scale projects, one significant milestone involved the comprehensive analysis of patient-level data for the EMA. This work continues to support post-authorisation studies, with plans for further impactful projects on the horizon.
Advancements toward qualification for the Global SMA Registry Network persist, building upon the encouraging support received in 2022. At the same time, our Global Registries Platform continues to expand its reach, with 10 registries using it for data collection and an upcoming enhanced version geared towards facilitating multilingual service and improved data quality.
TREAT-NMD Advisory Committee for Therapeutics
The TREAT-NMD Advisory Committee for Therapeutics (TACT) reviewed nine drug development programmes in 2023, providing expert objective advice to help to optimise their design and development. The committee met in Philadelphia in the spring where four programmes were reviewed and then the rest took place online. The meetings have resulted in productive discussions, culminating in advisory reports for participating companies. We are looking forward to holding two in-person meetings in 2024, one in spring in Amsterdam which is already full and then the Autumn meeting will be in Boston. If you’re interested in reserving a place or learning more, please visit the TACT section of our website.
Our commitment to global collaboration is evident in our engagement with the FSHD society in Project Mercury, which is aimed at accelerating treatments to FSHD patients globally. TREAT-NMD is leading on the two workstreams; developing registries and the FSHD dataset and launching targeted clinician and patient education programmes.
Throughout 2023 TREAT-NMD has led the development of a transformational project in response to European commission IHI call three. This has led to the successful signing of the grant agreement in November 2023 and the four-year project will commence 1st January 2024. Further details will be announced soon.
In the past year, TREAT-NMD achieved milestones such as hosting the DMD Early Diagnosis Summit and an impactful SOP workshop in Amsterdam. SOP content reviews are complete, and we anticipate a ‘Go Live’ date in Q1 2024.
Ongoing collaborations with industry partners involve contextualizing new treatments with real-world evidence, with a view to supporting regulatory decision making in the future.
In 2023 TREAT-NMD has established the Board of Trustees for TREAT-NMD Alliance Ltd and the NMD Advisory Committee (NMDAC) for TREAT-NMD Service Ltd. These bodies, include clinicians, researchers, patients, and elected experts, and play a crucial role by conducting a thorough assessment of the neuromuscular disease landscape. They will identify priority areas for focussing our future efforts. Their insights play a vital role in shaping our strategic direction, as guided by the newly formed Board of Trustees.
In reflecting on the year, we also honour the memories of those in the neuromuscular community who departed in 2023. Their legacy inspires our unwavering dedication to advancing treatments and enhancing care for individuals grappling with neuromuscular diseases. Heartfelt thanks to every individual whose dedication and hard work have made 2023 an exceptional year for TREAT-NMD and the global neuromuscular community.
As we near the close of 2023, we want to wish you a joyous holiday season and a promising New Year ahead
TREAT-NMD Chief Executive