TREAT-NMD Newsletter Survey

TREAT-NMD publishes a monthly newsletter featuring news on meetings, workshops, publications, studies and more from the NMD community. Readers can access the newsletter by email by subscribing to the mailing list, or by viewing it online on the TREAT-NMD website.

So that TREAT-NMD can continuously improve the newsletter and ensure its relevance to our readers we would be grateful if you could take a moment to complete our anonymous survey here. Your feedback is very much appreciated!

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TREAT-NMD Universal Registry Platform

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We are now halfway through the Universal Registry Platform (URP) Project and we are making good progress.

The Clinician Portal build was completed in mid-December 2020 and was ready for Beta testing as part of the project programme. The Beta testing was undertaken by a selection of DMD and SMA Registries, who were provided access to the system and began testing the week before Christmas. The Registries were asked to provide all testing feedback by the close of business on the 22nd January 2021 and that feedback is currently being collated, it will then be analysed internally by the URP project team before any change requests or additions to the system are proposed. Meetings will be held in early February to discuss the testing feedback in more detail to agree any changes and these meetings will be with the IT contractors, Dataset Project managers and TREAT-NMD Global Data systems Oversight Committee (TGDOC) Chairs.

The Patient Portal build began at the beginning of January and development is currently underway with updates being provided at the fortnightly ‘sprint’ meetings. As part of the Patient Portal build, we are also looking at Dual Reported Registries and gathering information to ensure that this element of the URP meets the needs of its users. We are anticipating being able to issue the Patient Portal for Beta testing by a selection of DMD and SMA Registries at the end of March.

In addition to the Clinician and Patient Portal for SMA and DMD disease modules, we have now received the confirmation of funding for adding a further disease module to the URP for Limb Girdle Muscular Dystrophy (LGMD). This is exciting news and as the build for the LGMD module will begin almost 5 months ahead of the intended project start date, it is a great show of confidence in the URP and will be of great value to the LGMD registries, many of whom do not have robust systems of their own.

On a final note, the URP team is growing and we will be welcoming two new team members at the end of January to assist with the planning, roll out and implementation of the URP across the TGDOC registry network.
For further information on the URP or to participate as a ‘test registry’ then please contact Caroline Ogden, the URP Project Manager or visit our website to learn more about the URP.

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Apply Now! Places Available on TREAT-NMD Virtual LGMD Masterclass 2021

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Apply Now! Places Available on TREAT-NMD Virtual LGMD Masterclass 2021

There are place still available on the TREAT-NMD Virtual LGMD Masterclass 2021. This masterclass was originally scheduled to be held as an in-person event in Barcelona, but it is now a virtual event and will delivered using our virtual conferencing software.

The event will be co-chaired by Professor Jordi Díaz-Manera, Professor of Neuromuscular Disorders Newcastle University and an Honorary Consultant Clinical Geneticist with the Newcastle Hospitals NHS Foundation Trust and Michela Guglieri, MD, a Senior Clinical Lecturer and Consultant Neurologist at Newcastle University and Newcastle Hospitals NHS Foundation Trust.

The programme, spanning 2 days will include 13 webinar lectures with opportunity for Q&A throughout. Day 1 features a range of webinar style sessions on diagnosis, cardiac care and much more. Day 2 features a choice of interactive workshops: Physiotherapy and other Clinical Care Considerations and Exemplary Clinical Cases. In addition to these sessions, delegates will be offered 5 pre-recorded lectures that will be made available in the run up the event as well as reading materials provided by the speakers.

We welcome applications from health care providers from across Europe who diagnose or manage the care of patients with Limb Girdle Muscular Dystrophy (LGMD), places are however strictly limited therefore we suggest applying early to avoid disappointment.

For further details about the masterclass, see our website, or you can apply for a place here.

If you have any questions about the masterclass or any education events at TREAT-NMD, please forward these to: education@treat-nmd.com

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New Turkish Translation of a Guide to the 2018 Standards of Care for DMD

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We are pleased to announce the guide to the 2018 standards of care for DMD is now available in Turkish. Thank you to the patient organisation DMD Aileleri Derneği and to Dr. Nazlı UÇUNOĞLU for all of their hard work in translating this fantastic resource so it can be used more widely. As well as the work on the SoC translations, DMD Aileleri Derneği are working on translating other useful resources for DMD patients. These articles can be found on their website.

The guide to the 2018 DMD SOC is now available in French, Portuguese (Brazil), Czech and Ukrainian as well as other languages, with more languages coming in the next few months. You can access them on our website here. If you require further information please contact: education@treat-nmd.com

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New Hebrew Translation of the DMD Guide for Families to the 2018 Standards of Care

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TREAT-NMD is delighted to announce that the DMD Guide for Families is now available in Hebrew. Thank you to the patient organisation Little Steps Association for Duchenne & Becker Patients in Israel and to the clinicians for translating this fantastic resource.

The guide is now available in US and UK English, Brazilian Portuguese, German, French, Ukrainian, Russian, Spanish and Czech. Further languages are in the pipeline and will be announced over the next few months. You can access them on our website here.

If you would like any of the guides printed professionally or require further information please contact our DMD Education Coordinator, Clare Bradley.

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New Issue of Journal of Neuromuscular Diseases Published

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A new issue of Journal of Neuromuscular Diseases (JND) has been published.

The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.

Volume 8, Issue 1 includes three open access reviews – (i) on peripheral neuropathies, (ii) on amyotrophic lateral sclerosis, and (iii) on glucocorticoid steroids in muscular dystrophy – plus nine research articles and two case reports.

To be kept up to date with all of the latest JND news, you can sign up to the journal’s newsletter here:

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New Mentoring Programme for Those Working in the Neuromuscular Field

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In 2020, European Neuromuscular Centre (ENMC) developed a new Mentoring Programme especially designed for mid-career scientists, clinicians and health care professionals working in the neuromuscular field.

The Mentoring Programme has been developed for people who wish to seek mentoring in order to become independent researchers and/or potential future leaders in the NMD field. These individuals will be at the stage in their careers where they are developing their own research plans and are establishing their research teams and collaborative networks.

In summary the programme consists of:
• Contact with two mentors, albeit in separate sessions, who have different and complementary backgrounds (i.e. academia vs business).
• Mentors and mentees are matched according to the type of skills that the mentee seeks to develop or improve.
• Mentors and mentee meet 2-3 times per year by video call, phone, online and/or if possible face to face.
• ENMC may help mentees to have access to the leadership training in business/administration
• Through its patient associations, ENMC may invite mentees to join one or two national patient days during his/her mentorship, taking place in the country of residence.

If you are interested in this programme contact Dr Alexandra Breukel

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Duchenne Ukraine International Conferences on the Treatment of Duchenne

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Duchenne Ukraine has conducted a series of international online conferences on the treatment of Duchenne patients.

The team consisted of participants from the Institute of Neurology of Schneider Children’s Hospital and Hadassa, Israel, Doctors from the Czech Republic and Belgium. More than 300 doctors, family members, and students of medical establishments registered for the conference from the Ukrainian. In addition, patients' organisations from Moldova, Russia, Czech Republic, Turkey, Kenya, Columbia, and Israel were present at the conference. The conference received support from the Regional Clinical Cancer Centre of Kyrovohrad, regional council and the National public TV company of Ukraine. Translation of video lectures and simultaneous interpretation was undertaken by the Duchenne Ukraine organisation, teachers of school No.6 and Donetsk National Medical University.

The conference topics were:
- Steroids and their side effects
- Establishment and work of the official registry
- Rehabilitation and physiotherapy

Videos of the conferences in English with subtitles in Ukrainian can be viewed on the YouTube channel "Duchenne Ukraine".

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