TREAT-NMD is thrilled to announce the launch of our brand new e-learning platform.  This fantastic interactive platform is aimed at healthcare professionals involved in the diagnosis and treatment of patients with neuromuscular disease.  You can expect to find free courses and resources in relation to:

• Limb Girdle Muscular Dystrophy including Patient Perspectives, Muscle Biopsy as a tool for diagnosis, Best practice for Cardiology and Respiratory management
• Standards of care for Duchenne & Spinal Muscular Atrophy

Learners will be able to lean at their own pace and access videos, quiz questions and other relevant and interesting resources.  Content is developed by renowned medical professionals, academics and  expert educators from leading universities and medical institutions around the globe.

You can access and signup up for a free account here.

We have a fantastic line up of speakers for the upcoming TREAT-NMD Conference which will be held in Vancouver, Canada from the 7th to the 9th of December this year.  One of which is Gita Ramdharry who is Consultant Allied Health Professional in Neuromuscular Diseases at the Queen Square/ MRC Centre for Neuromuscular Diseases. Check out the video below for a sneak preview of what Gita will be talking about at the conference.

View the full programme or register via our TREAT-NMD conference website.

We are now welcoming companies or academic groups to apply for advice on their therapeutic programmes from the TREAT-NMD Advisory Committee for Therapeutics (TACT).

TACT is a committee comprised of over 140 global experts in neuromuscular disease.  From this committee a bespoke panel will be put together to provide advice and guidance to de-risk your therapy development programme.  Applicants to TACT will receive a confidential, comprehensive review and a report containing recommendations emphasizing a rigorous, milestone-driven approach.  This in turn will enable optimal use of funding and resources. The result being programs which are less likely to encounter common pitfalls in development, and more likely to be funded or partnered.

The next meeting with availability will take place in Philadelphia from the 21st to the 23rd of April 2023.  You can find out more about the TACT process and benefits here and if you would like to set up a call to discuss how TACT can help you,  please contact laura.robertson@treat-nmd.com

The next in our series of educational masterclasses will be in Limb Girdle Muscular Dystrophy and is aimed at a North American audience.  The event will take place on the 3rd and 4th of November this year online and healthcare professionals involved in the diagnosis and treatment of patients with neuromuscular disease are welcome to apply.

The masterclass will contain talks and interactive workshops delivered by expert speakers in Limb Girdle Muscular Dystrophy including topics such as: standards of care; best practice; diagnosis and more!  You can view the programme  here. 

There will be plenty of opportunities to ask questions and to network with other delegates and speakers via our masterclass app.

Interested parties are required to pay a deposit of just £30 (not including platform administration charges) which is refundable upon masterclass attendance. To apply for your place, and for more information about all deposit T&Cs, view the masterclass application form.

Andoni URTIZBEREA, Institut de Myologie, Paris, France.

A number of countries are under-represented within the TREAT-NMD Alliance. The composition of the overall membership (around 1,000 people) and of the Executive Committee (EC) reflects this trait quite well.

Despite a growing interest in myology manifested by many neuromuscular specialists and patient advocacy groups from emerging countries over the past decade, their voice is hardly audible. Some of them joined various working groups of the Alliance just on the basis of cooptation.

Many of these professionals and advocates live and work in Low Middle Income Countries (LMIC) but not always.

Therefore, the EC decided to think about a task force susceptible to rectify this trend towards more inclusivity.

Three conference calls have been organized with health care providers that were already known and identified in the neuromuscular field, some already  TREAT-NMD members.

The first meeting took place on 18^th February and gathered participants from Central and South America, the second  gathered specialists from Africa, Middle East and Central Asia including Russia on 21^st February and last, but not the least, the third rallied people from the Asian-Pacific zone, held on 1^st March.

The goals and activities of the Alliance have been presented in a comprehensive way including the creation of this new task force.

The feedback has been largely positive. A few participants (from Chile, Iran and Africa) volunteered to join the forming group. It seems more challenging for colleagues located in Asia-Pacific due to time constraints but, after reading this note in the newsletter they might be willing to join as well. They are welcome.

The agenda and deliverables of the task force are still under construction. Among many areas of interest, it is planned to work on the governance, considering how individuals from these countries could be more involved in the groups and projects of TREAT-NMD and help further developments in the Neuromuscular field

The overall objective is a better recognition of parties both ways: To make the Alliance and its activities more visible in their own countries (by co-branding educational events, for example) and to provide them also with more representativity in the Alliance’s governing bodies.

If you are aware of individuals or organisations from these countries who are not a member of TREAT-NMD, and would have a keen interest in being involved with this agenda, please reach out to your networks and ask them to contact info@treat-nmd.com for ways to be involved.

 

Over the next few weeks, TREAT-NMD Services Ltd will be recruiting a Research Communications Lead to join our team. They will work with academic/clinical members of the network and TREAT-NMD committees to highlight the work being done within the network and we’re particularly looking for somebody with experience in rare NMDs. It’s a role that could be done remotely, or as part of a group elsewhere, so if you or somebody you know might be interested, please email:  info@treat-nmd.com.

Since 2009, the TREAT-NMD Advisory committee for Therapeutics (TACT) has served the neuromuscular community by providing independent and objective advice on the developmental pathway of  neuromuscular drug development programmes. Due to the success of TACT, staff from Newcastle University have developed a toolkit to replicate the TACT model in other disease areas.  The Advisory Committee for Therapeutics (ACT) toolkit provides step-by-step procedural advice for other disease communities, who share similar challenges in therapy development, to establish their own ACT. The toolkit was developed through the European Joint Programme for Rare Diseases (EJP RD)* and will soon be available for researchers to freely access in the EJP RD Innovation Management Toolbox (IMT). One of the goals of EJP RD is to identify good practices and support adoption to other communities, to help address the many unmet needs for people living with rare disease. Given the potential for the European Reference Networks (ERNs) to strategically oversee the creation of ACTs in different areas, an EJP RD-funded ERN training workshop was held in 2021 to introduce the model and the toolkit. Organisers of the workshop are now in discussions with some of the participants to discuss replicating the model through their ERN; for instance, the ERN for neurological disorders is exploring this activity at present.

In addition to their discussions with ERNs, Joanne Lee (Newcastle University) and Cathy Turner (TACT Project Manager) have provided advice to Dr Nicky Huskens, CEO of the Tessa Jowell Brain Cancer Mission, as they set up their own version of an ACT, known as the Brain Tumour Research Novel Therapeutics Accelerator (BTR-NTA). The BTR-NTA was set-up in collaboration with Tessa Jowell Brain Cancer Mission, Brain Tumour Research and NCRI (National Cancer Research Institute). Former TACT Chair Prof. Katie Bushby and now Vice-Chair of the Tessa Jowell Brain Cancer Mission, first introduced the TACT model to Nicky as one of the most successful and transformative programmes that she had been part of. Nicky became excited about the model and felt that a similar programme would be highly valuable in the brain tumour field. When asked about how the ACT model could be of benefit to their field, Nicky responded:

“There have been very few new drugs for brain cancer in the last decades. We hope that the ACT model will give scientists access to world class expertise so that they can continue their work with renewed focus. We hope the programme will help scientists think more broadly about their work and connect scientists with experts in relevant fields. We also hope that the programme will make it easier to obtain funding for promising therapies and we aim for the programme to assist scientists with more commercial elements as well. In time, we hope to see more new successful therapies in human trials.”

Nicky pitched the programme to the Mission’s Board and has since secured both funding (provided by Brain Tumour Research), approval and valuable input into the design and delivery of the programme. Members of Newcastle University and the TACT secretariat have provided them with advice in setting up their programme, which they have found to be incredibly helpful. Although their ACT is still in the early planning phase, they have had lot of support from their collaborating partners and other stakeholders within their community. They have  also obtained three year funding, recruited a programme manager to run the programme, recruited an oncology co-lead and are in the process of interviewing members for the committee. They have also advertised the programme to the community and have several potential applicants lined up.

If you would like more information about the ACT model and toolkit, please contact joanne.lee@ncl.ac.uk.

If you would like to find further information about TACT, specifically, please contact catherine.turner@ncl.ac.uk.

* This initiative has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575.

Muscular Dystrophy UK and the Adult NorthStar are holding an online symposium on the management of bone health and endocrine issues in adults with DMD on 31^st October from 10am – 1pm.

The symposium will aim to highlight standards of care in these areas as laid out in the recent Adult North Star publication but also discuss gaps in knowledge. The publication is a consensus document aimed at therapists who are working with adults with Duchenne muscular dystrophy with an aim to improve standards of care and can be viewed here.

Some of the topics covered in the symposium will include: Osteoporosis in DMD,  bone density assessment in adolescents and adults with DMD and puberty and gonadal function in adolescents and adults with DMD.  To  see the full agenda please click here and you can register for the event here.

MyFSHD, by Peter L Jones, PhD, is a bi-weekly podcast about education and personal empowerment for the worldwide facioscapulohumeral muscular dystrophy (FSHD) community. The podcast contains interesting discussions on many things of interest to the FSHD community. Learn about the science behind the different FSHD therapeutic approaches, FSHD pathology, family genetics and FSHD diagnostics. There are also discussions upcoming clinical trials and what to look forward to.

The podcast is available to listen to for free on Spotify,

A new issue of Journal of Neuromuscular Diseases (JND) has been published online. 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 9, Issue 5 has 10 articles, three of which are open access: a review covering late-onset Pompe disease; an article that looks at neuromuscular diseases affecting feeding, swallowing, and speech; and a paper about real-world adherence to nusinersen in adults with spinal muscular atrophy. Discover all the issue’s content here! To be kept up to date with all of the latest JND news, you can sign up to the journal’s newsletter at: http://tiny.cc/JNDsignup.

Issue link: https://content.iospress.com/journals/journal-of-neuromuscular-diseases/9/5    

Event: 3rd International FAIR Data ‘Visisting’ for Duchenne & Other Rare Diseases
Date: Tuesday 22nd November 2022
Time: 13:00 to 17:00 Central European Time
Format: Online

For the first time, the World Duchenne Organization will team up with RaDiOrg Rare Diseases in Belgium and SMA Europe in Germany to form a wider network of approximately 160 Patient Organizations all interested in how the FAIR principles could make a difference in optimising the use of patients’ data. Combined with the expertise of clinicians from ERN EURO-NMD, this small event consortium hopes to provide the broader Rare Diseases network, a better understanding of what is being implemented at the European level in regards to data ‘visiting’ across borders. The word ‘visiting’ will replace ‘sharing’ as with FAIR technologies data stay at source.

• Read on how patient organizations can drive FAIR data efforts to facilitate research and health care here
• Find out how a patient-led registry achieved a FAIR status by implementing an innovative FAIRification approach here