The aim of TREAT-NMD is to accelerate treatment and improve standards of care for people with neuromuscular disease. In order to best achieve this aim, TREAT-NMD has been reviewing and updating our workstreams over the past few months and we are pleased to share them with you in the diagram below.

We can support projects from the early preclinical stages through to market development in various different ways. Utilising our fantastic education programme and facilitating global registry enquires, we can support industry, patient organisations and healthcare professionals globally to ensure the most effective treatments and standards of care are received by those living with neuromuscular disease.

To find out more about what TREAT-NMD does or how to collaborate and get involved get in touch: info@treat-nmd.com

The 7th TREAT-NMD International Conference is fast approaching and we want as many delegates as possible to join us at the Vancouver Convention Centre between Wednesday 7^th and Friday 9^th December.  Spaces are now limited, but we are extending registration until Monday 21^st November for anyone who still wishes to attend!

The conference is not one to be missed – there will be presentations from world leading experts including Carsten Bonneman, Sharon Hesterlee, Robert Graham and Zoe Davidson as well as networking opportunities with representatives from over 70 organisations from across the globe!  Get registered and pencil the date in your diary today!

Register here and secure your ticket today!

The TREAT-NMD team were delighted to attend the 27th Annual Congress of the World Muscle Society. The congress took place earlier this month in Halifax, Canada and was an excellent opportunity for colleagues to catch up on global developments in the field of neuromuscular disease.

Several posters were presented highlighting the activities of the TREAT-NMD Network. These gave an overview of data within the FSHD and DM Global Registry Network, and presented cases where registry data has been used to inform regulatory decisions.

The conference also gave an opportunity to meet researchers, clinicians and other colleagues in person and preliminary plans for some exciting collaborations between network members were made. We’d like to say thank you to everybody that stopped by our booth and we’ll be in touch shortly to follow up. If you missed us, and want more information please contact info@treat-nmd.com or visit our website!

Since late last year, TREAT-NMD has been working towards EMA Qualification for our spinal muscular atrophy (SMA) core dataset and Global Registries Platform/Central Data Warehouse.

The Qualification process provides a regulatory opinion on a registry’s technology, processes and dataset. It assesses whether a registry demonstrates the ability to provide consistent, quality assured data that can support regulatory decision-making for a set of specified scenarios (e.g. post marketing surveillance or provision of natural history data).

Working closely with SMA stakeholders including clinicians, physiotherapists, patient representatives and registry curators, TREAT-NMD sought EMA advice covering our dataset and also our data management, data quality, and data security processes for a range of regulatory uses earlier this year.

After several meetings with the EMA, the final Scientific Advice was received from the EMA’s CHMP in September; this will be followed by a public letter of support from the EMA.

The Advice gives an in-depth response to our application and sets out a number of details that should be considered in our application for Qualification Opinion. This is the final step in the Qualification process, and we hope to address the points raised as soon as possible so an application can be submitted during 2023. This will create a resource that meets EMA requirements in SMA, and we are already working towards applications in DMD and LGMD as well.

The Neuromuscular Centre of Excellence was launched at the Christian Medical College and Hospital, Ludhiana on the 28^th February 2022 as part of the celebration of World Rare Diseases day. The aim of the centre is to provide comprehensive care: from awareness and advocacy, to diagnosis and treatment and training and research focused on neuromuscular disorders.

Established in 2016, following a protocol-based approach built on international standards of care, specialists at the Neuromuscular CoE offer comprehensive workups to achieve accurate diagnosis of nerve and muscle diseases and rely upon state-of-the-art treatment modalities to optimize quality of life.

In February the centre organized a city-wide motorcycle rally on World Duchenne Awareness Day. More than 60 bikers including city administrators rode for the cause and students from five colleges participated in spreading awareness by hosting a carnival on campus and a cultural event including a street play in the city square.

There is also a new Physical Medicine and Rehabilitation room at the centre equipped with resources for physical therapy and occupational therapy and a facility for advanced muscle biopsy. The new facility will have capacity for fresh frozen muscle processing and enzyme histochemistry and immunohistochemistry which will help with difficult cases and research.

You can find more information on the centre, what they do and how to get involved via their website.

Earlier this year, Muscular Dystrophy UK (in association with the Adult NorthStar Network) has funded and produced a collection of videos that provide an easy-access guide outlining the standards of care for adults with Duchenne.

The bite-size videos feature leading specialists in neuromuscular care and provide an accessible resource for patients to present to their GPs and other non-neuromuscular specialist health professionals involved in their care.

The video collection follows the first ever Consensus Guideline for the Standard of Care of Adults with Duchenne Muscular Dystrophy which was published in the Journal of Neuromuscular Diseases in October 2021.

These Standards of Care were developed by the Adult NorthStar Network to help drive improvements in services and set national care standards for adults living with Duchenne Muscular Dystrophy. MDUK has funded and supported the Adult NorthStar Network since its establishment in 2016.

The videos present guidance from leading specialists in the following areas:

• respiratory care
• cardiac care
• renal and bladder
• gastrointestinal care and nutrition
• general anaesthesia
• bone health
• the role of neuromuscular care advisors
• psychological wellbeing
• palliative care
• physiotherapy

The Adult NorthStar Network also published a therapist-specific guideline in May 2022 to support physiotherapists and other therapists who may have little or no experience in managing these complex care needs.

Earlier this month, The World Duchenne Organization launched the Accredited Duchenne Centres Programme. This global program is part of the unique development of a worldwide accreditation for Duchenne Centres. It consists of an ongoing development of the process of criteria and visitation leading to the right to be named an Accredited Duchenne Centre.

This Program will not only accredit Duchenne Care Centres providing multidisciplinary care according to the latest Standards of Care and Care recommendations, but will also provide training regarding optimal Duchenne care and support for Duchenne centres which cannot yet fulfil the criteria for accreditation.

To find out more and read the full press release here.

The 9th annual congress of the European Academy of Neurology will take place in Budapest from 1-4 July, 2023.

The Congress will provide a fantastic chance for delegates to come together and establish new connections as well as catching up with old ones.  Themes include Optimising the use of digital health technologies and telemedicine, Artificial intelligence: advances and applications in neurology and using data science to transition to an era of precision health.

Deadline for abstract submission will be 12^th January 2023 and, if you would like to benefit from early registration, please visit the EAN website and register by 26^th  April 2023.