Welcome to our TGDOC summer newsletter where you can find all the latest dataset and Global Registry platform updates, updates from disease subgroups, events, publications, and much more all in one place.
TGDOC New Members
A very warm welcome to our TGDOC new members:
- Dutch LGMD Registry
- Swedish National Registry for Neuromuscular Disorders (NMiS)
- Romanian DMD/DMB Patient Registry
- Telethon Registry of Neuromuscular Diseases (Mexico)
We strongly believe that your presence will enrich our knowledge in Neuromuscular Diseases sphere.
We are also working with a number of registries around the world to facilitate their membership. This includes registries in Mexico, Ukraine, Japan, Spain, Pakistan and a range of South American countries.
If you require additional support with this process, please contact email@example.com.
TGDOC Annual Curators Meeting
You may have received an email with regards to the TGDOC Annual Curator’s meeting (ACM) that will take place in conjunction with the TREAT-NMD Conference in Vancouver, Canada. The details of the ACM and support for curators to attend are outlined as below:
Schedule of Events:
- Thursday 8th & Friday 9th December – TREAT-NMD Conference
– key areas in neuromuscular diseases: Patient Engagement, Clinical Trial Considerations, Global Neuromuscular Care and access to treatment and more.
- Saturday 10th December (Morning) – Annual Curators Meeting (hybrid)
– content to be announced later in the year but designed by curators for curators. We are very keen to hear from curators what they would like to see included. Please contact firstname.lastname@example.org with any ideas. We are always open to registries leading on topics too.
What TREAT-NMD can provide to support registry curator attendance at the above events:
- Complimentary ticket to attend the TREAT-NMD conference
- We will contribute towards economy flight bookings (£500 for North American flights, £750 for Europe and £850 for flights from elsewhere) and £30 for airport transfers in Vancouver.
- We will provide hotel accommodation for Wednesday, Thursday and Friday
- Breakfast and lunch whilst you are in Vancouver
- Dinner on Thursday evening
- Attendees will be responsible for dinner on the Wednesday and Friday and all other expenses
Please note the above is available for one representative per registry. We would appreciate it if you could please complete the following form by the 26th August 2022 registering your interest in attending. Thank you.
The TREAT-NMD 7th International Conference
The 7th International TREAT-NMD Conference will be a fantastic opportunity for patients, academics, clinicians, patient registry curators and industry representatives, to get together to network, learn and exchange ideas about translational research.
Some topics that will be covered include: patient engagement; Clinical trial considerations in ultra-rare patient groups; Global Neuromuscular Care and Access to Treatment; Emerging Therapeutics options in NMD and many more! View the full programme here.
This fantastic in person event will take place from the 7-9th December 2022 in Vancouver, Canada and there will be lots of opportunities for discussion and networking. At our conference you are not just part of the audience, you are part of the discussion!
Tickets: Reserve your ticket NOW!
Abstracts: Open Now! Closing date: 7th September 2022. Submit your abstract here.
TREAT-NMD Team Update
We’re sad to announce that Ben Porter is leaving TREAT-NMD at the end of July. Neil Bennett will be taking over as Global registry manager. Neil has been at TREAT-NMD since early 2022 and has experience in patient registries from his previous job.
We are, however very pleased to announce several new additions to our growing team – Hsin Chieh Chua joined the Registries team on June 7th as Global Registry Network Coordinator, and she will lead on global registry enquiries. Farjana Ali, who joined TREAT-NMD in May will be working with the registry team to support the rollout of LGMD and DMD datasets and TGDOC membership.
Jess Page joined the SMA dataset team in July as the SMA Core Dataset Project Co-ordinator.
Emma Watson and Seung Yeon Lee both joined as project managers and Nathan Bent as our new Data Governance Manager. Nathan will be ensuring that our policies and governance meets all necessary regulations.
LGMD and DMD Dataset Project Update
The Duchenne Muscular Dystrophy (DMD) and Limb Girdle Muscular Dystrophy project teams continue to engage with and support registries to progress the adoption of the new datasets. There are eight registries who have adopted the DMD dataset in their existing platforms and five have adopted the dataset via the Global Registry Platform.
The latest version of the DMD dataset (V1.2) has 112 unique data items (73 mandatory & 39 non mandatory items for clinically reported registries ) and has been curated with the guidance of global leading experts.
Working closely with the TREAT-NMD project team, the Czech REaDY registry became the first registry in the Europe to implement the new LGMD dataset (v1) into their platform & started collecting the new dataset in May.
Lívie Mensová, Expert guarantor of the LGMD registry commented:
“… The LGMD dataset rollout has been an extension of the previous DMD & SMA dataset implementations, the process has been refined with every new dataset rollout. It has been created with guidance from key opinion leaders and the quality of the dataset is evident. The collection of this vital data will aid in the development of future clinical projects. As always, it has been great working with the TREAT-NMD team, and we look forward to working with them for future projects.”
Congratulations to Lívie Mensová, Expert guarantor of the LGMD registry, Lenka Mokrá the Project manager and all the Czech REaDY Registry team. You can view the registry data for the Czech REaDY registry via this link: Czech REaDY Registry Data Overview.
If you have received emails from the datasets projects team, would like to discover more, and are yet to engage please contact email@example.com and we will set up a Zoom call at a mutually convenient time to discuss your registry’s needs and what we can do to support you in the adoption of the dataset. The support we can offer includes:
- Local language interpretation on calls if registries would find this more convenient
- Gap analysis between current dataset & V1 to understand the scope of the work
- Advice and guidance on an appropriate approach to project planning & implementation
- Liaison with other registries who have implemented the dataset to share their experiences
- Financial support to help registries with their implementation activities & data collection
The team look forward to meeting registry curators in person at some of the conferences and events planned for 2022 where we can discuss collaborating to adopt the new dataset, either through implementing it into your existing registry platform or by adopting the Global Registries Platform or GRP
SMA Dataset Project Update
As you may know, TREAT-NMD significantly expanded the SMA core dataset between 2018 and 2020, and all TGDOC member registries are now asked to comply with SMA Core Dataset v.2, or at least begin working towards it. This is the final year of the Expanded Dataset Implementation Project which has supported over 30 registries globally to adopt the core dataset. We would strongly encourage any SMA registries who have not yet joined the project to take advantage of the support offered while there are still spaces available. There is a range of support available which includes:
- A bursary of up to €8000 is available to eligible registries, in recognition of the work needed to implement the SMA Core Dataset.
- Access to a network of registries and curators who have already implemented the SMA Dataset.
- Monthly project support drop-ins to access information and guidance from the project team.
- Annual SMA Dataset Workshop – this year it will be held in Vancouver (7th December 2022)
- Additional resources available on the project webpage.
- Direct linkage with other TREAT-NMD Dataset projects (DMD and LGMD).
If you would like to get in touch to discuss this further, please contact Julie Bohill, SMA Dataset Expansion Project Manager (Julie.firstname.lastname@example.org). More information about this project is available here.
Since the start of this year the team at TREAT-NMD have worked with the TGDOC Chairs, subgroup leads and curators to submit abstracts to several conferences around the world. In June, posters were presented at the Cure SMA Research and Care meeting (view SMA Poster), FSHD Society International Research Congress ( view FSHD Poster), the International Congress on Neuromuscular Diseases and a talk and two posters at the International Myotonic Dystrophy Consortium Meeting (view talk and view poster)
Later this year, the global registry network will be represented at the Muscle Study Group Meeting and WMS – please do stop by if you’re at one of these meetings.
2023 Myotonic Dystrophy Foundation Early Career Research Grant RFA
This year the Myotonic Dystrophy Foundation (MDF) intends to issue up to two, two-year awards of up to $380,000 at $190,000 per award for early career scholars in the DM field for projects that focus on basic, translational and/or clinical research or care projects in myotonic dystrophy. The MDF recognizes the importance of recruiting and retaining basic science early career researchers, as well as the critical need to recruit and retain clinical early career researchers to focus on the field of DM.
Applications are due by September 9, 2022, with a start date for successful applicants of January 2023. Applicants are encouraged to contact MDF’s Chief Executive Officer, Dr. Tanya Stevenson, with any questions about this RFA or the scientific content of their proposals. Technical issues should be directed to MDF’s Research Coordinator Dr. Nadine Ann Skinner.