Reflecting on 2024 and Celebrating a Year of Progress and Collaboration
With deepest gratitude, we extend our thanks to the incredible individuals who have generously shared their expertise, guidance, and precious time, driving forward our mission to transform the diagnosis, care, and treatment of those living with neuromuscular diseases. TREAT-NMD’s work would not be possible without the incredible contributions of our industry partners who fund many of our projects —thank you for being a part of this journey.
As 2024 draws to a close, we reflect on a year filled with remarkable achievements, impactful events, and milestones that have furthered our mission. From advancing groundbreaking data projects and hosting our first Becker patient events to celebrating 15 years of TREAT-NMD’s Advisory Committee for Therapeutics (TACT), and supporting registries worldwide, this year has demonstrated the power of collaboration, innovation, and dedication.
In this roundup, we’ll share highlights from our Projects Team, Registries Network, Education initiatives, Data Governance advancements, and more. These accomplishments reflect the collective efforts of our network and set the stage for continued progress in 2025.
Here’s to celebrating a year of success and moving forward together!
Reflecting on a Remarkable Year for TREAT-NMD’s Projects Team
2024 has been a year of exceptional progress for the TREAT-NMD Projects Team. Key achievements included the completion of Several data projects that have advanced understanding of the natural history, care pathways and long-term treatment impacts in Spinal Muscular Atrophy (SMA).
The introduction of a groundbreaking Duchenne muscular dystrophy (DMD), Early Diagnosis Framework and the initial draft of the Limb Girdle Muscular Dystrophies (LGMD) Family Guide represent major advancements in providing support to patients, families, and caregivers around the globe. Visit our resources page to explore these and other tools available for the neuromuscular community.
Operationally, we successfully integrated a full Project Management Office, enhancing our project tracking, resource and capacity planning, and overall delivery efficiency for our partners and stakeholders. This transformation has laid the foundation for even greater impact in the years to come.
Looking ahead to 2025, we are focusing on data quality initiatives, the development of new data projects, and updating our Standard Operating Procedure (SOP) Library, available on the TREAT-NMD website. These efforts will further strengthen our commitment to improving care and outcomes for neuromuscular disease communities globally.
Exciting Milestones and New Initiatives for the TREAT-NMD Global Registries Network in 2024
The TREAT-NMD Global Registries Network has had an eventful few months, achieving significant milestones and setting the stage for exciting initiatives in 2024. We welcomed four new registries to the network, bringing the total to 70, further expanding our global reach and strengthening collaboration in the neuromuscular community.
A major achievement lead by TREAT-NMD Global Data systems Oversight Committee (TGDOC) has been the development of a draft “standard neuromuscular dataset,” aimed at simplifying core dataset components across all neuromuscular disorders for different use cases. This dataset is already receiving valuable feedback and is a crucial step toward harmonising data practices across research and clinical settings. We will be presenting this dataset proposal to our curators at the next curators meeting and are looking forward to adding this work to TREAT-NMDs datasets early next year.
In terms of international engagement, our participation in the European Medicines Agency (EMA)/Heads of Medicines Agencies (HMA) Registries Workshop in February provided an excellent opportunity to engage with global experts, share our progress, and align with regulators on best practices. Additionally, we had a strong presence at key conferences like International Myotonic Dystrophy Consortium (IDMC-14) and World Muscle Society (WMS), presenting scientific posters and furthering collaborations with others in the neuromuscular space.
One of the key focuses of TREAT-NMD this year, and moving into the next year has been data quality. We have developed new tools designed to assist registries in monitoring and diving deeper into aspects of data integrity. This goes towards ensuring the data collected by our network is reliable and suitable for various use cases. A key piece of work for 2025 will be the development of the TGDOC data quality plan. This is an important initiative to elevate global data standards in keeping with regulatory recommendations.
Visit our Global Registries Network page to learn more.
Breaking New Ground with the Myotonic Dystrophy (DM) subgroup Publication on the Largest DM2 Cohort Analysis
Our DM subgroup have worked hard this year on a release of a new publication in the Journal of Neuromuscular Disease spearheaded by our DM subgroup lead Stojan Peric. This peer-reviewed article represents patients from network member DM registries and has resulted in an analysis of the largest DM2 cohort assembled to date. The paper provides demographic and clinical data for future research and trial recruitment and illustrates TREAT-NMDs international reach and the importance of capturing DM2 data.
We would like to extend our thanks to all the curators and authors for taking part in this project, with a hope that this will lead to more publications in other NMDs soon. For the full paper, please contact us if you would like to see a copy.
Advancing Education and Global Collaboration
A significant step for the Education & Events team was hosting TREAT-NMD’s first-ever Masterclass in the MENA region in May 2024. This three-day event brought together leading experts, healthcare professionals, and researchers from 21 different countries to share cutting-edge insights into DMD, SMA, and gene therapy. It provided an exceptional platform to connect, educate, and further global understanding of these conditions. This Masterclass was a key part of our ongoing mission to expand our reach and foster collaboration worldwide.
Whilst we were disappointed to postpone the TREAT-NMD International Conference, originally scheduled for February 2025 in Dubai, we remain committed to advancing our efforts in the MENA region.
To ensure an exceptional experience for all attendees, we’re excited to announce that the new date and location for the conference will be shared in early 2025. We look forward to welcoming everyone to an impactful event!
Uniting the European Becker muscular dystrophy community
On 14th September 2024, TREAT-NMD hosted its first-ever simultaneous patient events for the Becker muscular dystrophy (BMD) community. With support from patient organisations, we brought together those living with Becker and their families from across Europe, to Amsterdam and Milan.
Participants accessed expert-led sessions on multidisciplinary care, genetic counselling, psychological support, transitioning to adult services, and emerging therapies, with live translations in five languages.
Beyond the presentations, networking and discussions created a safe space for attendees to share experiences, exchange advice, and form meaningful connections. Seeing patients support one another and guide families of younger individuals highlighted the strength of this community.
We look forward to continuing to educate the neuromuscular network in 2025, kicking off with our DM1 virtual masterclass in January, for health care professionals.
Learn more about our Masterclasses and upcoming events on our Education page.
Marking 15 Years of TREAT-NMD’s Advisory Committee for Therapeutics (TACT) with Our Inaugural Symposium
On 31st October 2024, TREAT-NMD marked a significant milestone with the TACT Symposium, Developing Therapies in Rare Neuromuscular Diseases, held in Boston. This event brought together experts, researchers, clinicians, and key stakeholders to reflect on 15 years of progress in developing therapies and improving patient outcomes.
The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a global team of experts providing confidential, comprehensive reviews to optimise drug development for neuromuscular diseases. Their constructive feedback has been pivotal in advancing therapies, and we encourage organisations to get in touch to learn how TACT can support their work. Discover how TACT can support your work by visiting our TACT section on our website.
The symposium highlighted key achievements, including advances in preclinical research, the critical role of patient perspectives, and the development of robust, patient-centric clinical trial measures. It underscored the need for continued collaboration to overcome challenges and drive innovation.
Looking ahead, TACT meetings are planned in Athens and Atlanta in 2025, offering further opportunities for organisations to collaborate and benefit from TACT’s expertise in advancing therapies for rare neuromuscular diseases. If your organisation is interested in learning how TACT can help, please get in touch with us.
Data Governance and Technology Team Celebrates Key Achievements
Over the past 12 months, the Data Governance and Technology team has achieved several important milestones, making great strides in supporting the global TREAT-NMD network. Here’s a look at some of our key accomplishments:
Global Support for Member Registries
We’ve continued to provide tailored Data Protection and Consent modelling and documentation, ensuring that TREAT-NMD member registries around the world have the support they need to operate efficiently and ethically.
Quality Assurance of Project Data
Our team has overseen the Quality Assurance of data provided by registry members, ensuring all Consent and Ethical Approvals are in place. We’ve also worked closely with the TREAT-NMD Registries Team to ensure that the data is properly Quality Controlled and meets the highest standards.
Welcoming Our First Data Engineer
In an exciting development, we’ve welcomed our first Data Engineer to the team. They are currently redeveloping our Central Data Warehouse to simplify and streamline the way we collect and organize data. The first phase of this project was successfully completed in November 2024, and further development is planned for Q1 2025.
Version 2 of the Global Registry Platform
We’re also making great progress on version 2 of the Global Registry Platform, which is used by several registries. The pilot version will be live on December 20, 2024, with initial testing beginning, and the full launch set for January 2025.
Our central data warehouse also underwent a significant rebuild, providing enhanced capabilities for future data projects.
Explore our work and tools by visiting the Data Governance Page.
TREAT-NMD Neuromuscular Disease Advisory Committee (NMDAC) – Enhanced Network Engagement in Strategy.
We are excited to announce the formation of the TREAT-NMD Neuromuscular Disease Advisory Committee (NMDAC) this year. As a key strategic advisory body for the TREAT-NMD network, the NMDAC plays a vital role in shaping the future of neuromuscular disease (NMD) research and care. Their primary objectives include identifying specific areas of unmet needs within the NMD field, creating task forces to tackle these challenges, and guiding projects where TREAT-NMD can make the most significant impact.
With a global representation, the NMDAC focuses on key diseases such as Spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), Facioscapulohumeral muscular dystrophy (FSHD), Charcot-Marie-Tooth (CMT), Limb-girdle muscular dystrophy (LGMD), and Myotonic dystrophy (DM). The committee is dedicated to identifying critical gaps in research and care, providing actionable recommendations, and helping TREAT-NMD prioritise initiatives that can drive meaningful change.
The NMDAC will meet quarterly, disseminating its findings through white papers and scientific conference presentations. Stay tuned for updates on their initiatives by visiting our NMDAC page.
Celebrating One Year of Project PaLaDIn
Since its launch in January 2024, Project PaLaDIn has achieved significant milestones, built strong partnerships, and laid a solid foundation for the future. The journey began with a kick-off meeting in March 2024 in Rome, where project partners and stakeholders aligned on goals and objectives, setting the stage for success.
Over the past year, we established the governance arrangements, funding streams, the industry engagement Interactium Futures Board (PIFB), and have held several Communications workshops. In early November, PIFB gathered in Boston to analyse the drug development pipeline and tease out the needs of the different stakeholders moving forwards and this will result in a white paper capturing key insights and outcomes, which will be shared with the wider community in early 2025.
Most recently, on December 17th and 18th, we hosted workshops addressing critical topics such as consent, permissions, and security. These sessions provided valuable opportunities to gather expert feedback and refine our approach.
Stay updated with the latest developments, announcements, and event details by visiting the official Project PaLaDIn website.
Looking Ahead to 2025
As we step into 2025, we pause to remember those in the neuromuscular community we lost this year. Their impact continues to resonate, inspiring us to push forward in advancing treatments and improving care for those affected by neuromuscular diseases.
We extend our deepest gratitude to everyone who has contributed to making 2024 a remarkable year for TREAT-NMD and the global neuromuscular community. Your dedication fuels our collective progress, and as we look ahead to 2025, we remain steadfast in building on this momentum to create a brighter future.
As we near the close of 2024, we want to wish you a joyous holiday season and a promising New Year ahead
David Allison,
TREAT-NMD Chief Executive
