Newsletter June 2022

The programme for the TREAT-NMD 7th international conference has now been finalised! The conference will take place from the 7th to the 9th of December in Vancouver, Canada this year and the final version of the programme can be viewed on the conference website. We look forward to sessions on patient engagement; clinical trial considerations in ultra-rare patient groups; global neuromuscular care and access to treatment; emerging therapeutics options in NMD and more across the two days.

The conference is aimed at patients, academics, clinicians, patient registry curators and industry representatives and will be a fantastic opportunity to network and acquire the latest updates from the neuromuscular community.

Tickets: Reserve your place NOW!

Abstracts: Open Now! Closing date: 7th September 2022.

These are exciting times at TREAT-NMD as we continue to expand the organisation and the team.  Farjana Ali joined TREAT-NMD in May as our new Project Administrator.  Previously Farjana worked at Northumbria University as a Department Administrator in the Faculty of Arts, Design & Social Sciences and she will be supporting the development of the LGMD and DMD datasets as well as providing support on other projects.  Hsin Chieh Chua joined the team in June 7th as Global Registry Network Coordinator. Hsin recently completed her MSc Business with international management at Northumbria University and prior to that worked with various transplant programs within the public healthcare sector in Singapore

In addition to this we are delighted to announce that Jess Page will be joining  the SMA dataset team in July as the SMA Core Dataset Project Co-ordinator. Jess recently completed her MRes in neuromuscular Disease at the John Walton Centre, Newcastle and has a keen interest centred around improving quality of life and health outcomes for those living with rare neuromuscular disease.

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 begin working towards it. This is the final year of the 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 €8000, available to eligible registries, to support implementing 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 (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.bohill@ncl.ac.uk). More information about this project is available here.

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  farjana.ali@treat-nmd.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

AGADA Biosciences workshop, “Rigor and Reproducibility in Murine Models of Neuromuscular Diseases for Preclinical Drug Development” has been postponed.  The workshop was due to take place on October 10th – 11th, 2022 in Halifax, Nova Scotia, Canada.

A new date for the workshop is yet to be announced.

Applications to AcadeMYO are now open! AcadeMYO is the digital version of the renowned Summer School of Myology.  The course has been running yearly since 1998 and will contain 40 hours of lessons from 60 experts in the field.  Taking place on the 11th, 12th and 13th of July it will be a fantastic opportunity to perfect your skills in myology, an increasingly appealing medical specialty dedicated to muscle disorders.

All professionals manifesting some interest in the field are encouraged to apply. Be prompt in doing so as the maximal gauge has been set to 100 participants.

The event programme is available to view online and you can apply here.

European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN EURO-NMD) have worked alongside the European Academy of Neurology (EAN) have created a free webinar series.  Recent topics include:

• Primary Mitochondrial Myopathies
• Duchenne Muscular Dystrophy Genetic Diagnosis
• Muscle Ultrasound, a biomarker tool for muscular dystrophies

Webinars can all be viewed on EURONMDs YouTube channel