TREAT-NMD Services Limited are thrilled to announce a three-year global partnership with Aetion, Inc., the global leader in real-world evidence (RWE) technology and analytics. The partnership will enable us to work with Aetion on a number of projects to expedite the development of treatment solutions for people with rare neuromuscular diseases.

These projects will combine the expertise of TREAT-NMD Global Registry Network that collects de-identified data from patients across the world with Aetion’s regulatory-grade expertise in RWE study design and analysis.

“TREAT-NMD is pleased to partner with Aetion on a range of projects to generate the scientific evidence necessary to develop and deliver novel treatment solutions to people with neuromuscular diseases around the world,” said David Allison, TREAT-NMD CEO. “We will work in concert to ensure that researchers have access to rare disease data while also leveraging Aetion’s trusted RWE capabilities.”

As globally-recognized partners, TREAT-NMD and Aetion strive to drive impact by speeding the delivery of rapid and robust RWE for the highly challenging area of rare neuromuscular disease. This collaboration, in turn, will support solutions for biopharmaceutical researchers by enabling easier access to critical rare disease data sources and scientifically-validated study design guided by first-in-class RWE methodologies.

“Aetion is inspired by TREAT-NMD’s mission to operate a collaborative, inclusive global network and data infrastructure to advance the diagnosis, treatment, and care for neuromuscular patients globally,” said Nicolas Deltour, VP of Real World Solutions at Aetion. “The richness of the TREAT-NMD registry network combined with Aetion’s RWE analytics technology and expertise will together play a pivotal role in supporting pharmaceutical companies in advancing treatments and better outcomes for neuromuscular patients in Europe and around the world.”

You can read the full press release here.

TREAT-NMD are delighted to be hosting a series of engaging, in-person masterclasses between 22-26^th May 2023 across three core subjects: DMD, Gene Therapy and SMA.

The event, which is being held in Amsterdam, will feature a series of 1.5 day masterclasses dedicated to each core subject and will provide delegates with the most up-to-date information around diagnosis, patient perspectives, standards of care and emerging therapies. Please see the TREAT-NMD Expert Masterclass Week Programme for more details regarding session content and speakers.

Applications are open and to ensure this event is accessible for all, TREAT-NMD will provide:

• A free programme of sessions led by expert clinicians, academics and practitioners
• Free accommodation*
• Contributions towards travel costs*
• Lunch and refreshments across the masterclass

Delegates are welcome to spend the whole week with us and attend all three masterclasses or select just one or two areas of interest.

Places are limited and applications are filling up fast so to avoid disappointment, apply today to secure your place.

*Please see the TREAT-NMD Alliance Ltd Travel and Expenses Policy for further details.

If your organisation is planning an educational event about a neuromuscular disease, you could apply to TREAT-NMD’s Educational Committee (TEC) for endorsement for your educational event.

TREAT-NMD offers endorsement for a range of educational events on neuromuscular diseases. The endorsement is a kitemark which can be awarded to a range of educational programmes including live events, e-learning courses and blended learning sessions. The kitemark demonstrates that your education programme meets a high standard and provides assurance to your attendees of the calibre of your course.

All applications are reviewed by the Education Committee TEC (TREAT-NMD Education Committee), a board of experts (academics and patient representatives) who are affiliated with TREAT-NMD and have an interest in the education events and materials that TREAT-NMD produces. Once the application has been reviewed, a decision will be sent to the applicant.

You can find further information about TREAT-NMD Education Committee Endorsement and apply for endorsement on our website.

Please email: education@treat-nmd.com  if you have any questions or would like further information about the endorsement process.

TREAT-NMD held a meeting in Amsterdam on the 9th and 10th of February 2023 to discuss and decide upon an updated dataset that will be collected by members of the TREAT-NMD Global Registry Network to support Post Marketing Surveillance (PMS) requirements.

A broad spectrum of Key Opinion Leaders and stakeholders from across the DMD field attended including, clinicians, physiotherapists, data experts, patient and patient organisation representatives, as well as pharmaceutical companies and medicines regulators and payors from US, EU and UK.

The group came together to determine the mandatory items to be included in a revised DMD Dataset for collection in relation to PMS for DMD, taking into account gene therapy and other drivers so registries will be prepared to collect real-world evidence relevant to post-marketing studies before designs are finalised and studies start.

The meeting explored the relevance of the dataset in the current landscape, the experiences of registries, clinicians and patients in collecting data, and the quality of data being collected. Requirements of pharmaceutical colleagues, regulators and payors were also covered. An implementation roadmap was agreed to address next steps and work will now progress to develop a PMS-ready registry network and dataset for DMD.

Hindi & Punjabi Translations of the Duchenne Muscular Dystrophy Guide for Families are now available!

We are delighted to announce that the DMD Guide for Families is now available in both Hindi & Punjabi languages. The guides are based on the 2018 Standards of Care for Duchenne Muscular Dystrophy (DMD) and provide valuable information to families and patients about aspects of care and treatment.

The translation of this guide into Hindi & Punjabi languages is courtesy of Mr Shubham Singla, Mrs Gagandeep Kaur, Dr Anusha Singhania and Dr Vineeth Jaison from the Christian Medical College & Hospital, Ludhiana, Punjab, India, and we are delighted that this brilliant resource can be used more widely.

The DMD guide for families is available in a wide variety of other languages including US and UK English, Brazilian Portuguese, German, French, Ukrainian, Russian, Spanish and Czech with more languages coming. You can view the DMD guides on our website.

For more information about the DMD Guide for Families, or to offer your support in translating our guides to other languages, contact: education@treat-nmd.com

Longstanding member of the TREAT-NMD Registry Network, the Myotubular & Centronuclear Myopathy (MTM & CNM) Patient Registry has grown from strength to strength since it’s foundation in 2013. Originally started by the patient organisation Myotubular Trust, it was transferred to the John Walton Muscular Dystrophy Research Centre in 2015 and is now a compelling example of the power of community collaboration in neuromuscular disease.

Jointly funded by the Myotubular Trust, MDUK, and Astellas Gene Therapies, the registry is governed by an independent Steering Committee and collects genetic and clinical data via an online portal from patients and clinicians worldwide.

One of the most recent developments improves the global accessibility of the registry by adding a tenth language to the translations available. From the end of March 2023, patients and families will be able to read the registry information and complete their forms in Arabic, in addition to the previously available languages of Dutch, English, French, German, Hindi, Italian, Polish, Portuguese (Brazilian), and Spanish.

Another new development proves that as well as collecting its own longitudinal data, the registry can serve as a vital data collection tool for standalone research studies. The registry is partnering with Canadian researchers from The Hospital for Sick Children (SickKids) in Toronto and Montreal Children’s Hospital to provide recruitment support and data collection services for an MTM Brain MRI Study. This study aims to determine the prevalence and type of neuroimaging abnormalities detectable by brain MRI in individuals with XLMTM. Using the registry in this way reduces duplication of data and effort, ensures appropriately controlled data access for the whole disease community, and minimises the burden of research participation on patients and families. The study will become active in March 2023 and will be recruiting male patients with XLMTM who have had a previous brain MRI.

If you would like to know more about the registry or explore ways of working together, please contact us using the details on our website.

If you have any patients for whom this is relevant, please let them know about the registry and encourage them to visit the website to find out more and register.

The 25th Summer School of Myology will take place at the Institute of Myology in Paris from June 12 to 16, 2023 in two ways: face-to-face, for 40 to 50 people, and online, on the same dates, for remote participants.

The full face-to-face course will include five full days of instruction combining lectures and practical workshops. Internet users will benefit from three interactive sessions of 4 hours each on Monday afternoon, Wednesday afternoon, and Friday afternoon.

One month before the start of the School and until September 15, 2023, all registrants (face-to-face or distance learning) will have online access to some sixty pre-registered courses.

All registrations will be done on the AcadeMYO platform after validation of the requests by the Scientific Committee of the Summer School.

Requests for prior validation should be sent by email to J. Andoni Urtizberea at the following address: andoni.urtizberea@gmail.com. A simple CV will be sufficient.

The payment of the registration fees (500 euros for a face-to-face participation, 250 euros for a remote participation) will be done on the AcadeMYO platform in a secure way.

The FSHD Society  has created a state-of-the-art, on-demand CME course for clinicians involved with the diagnosis and management of care for patients with facioscapulohumeral muscular dystrophy (FSHD).

The course presents information on the latest in diagnosis, genetic counselling and testing, natural history, symptom management, and standards of care. This course also provides a forum for thought leaders to share ideas, challenges and successes in delivering optimal care to people with FSHD. Those taking the course will learn about the current therapy development and clinical trial landscape and the emerging implications for care. If you are interested in signing up or learning more click here.

The 4th Congress of the Latin American Society of Neuromuscular Diseases (SOLANE) will take place on the 1^st-3^rd of June 2023 in Bogota, Colombia. This fantastic 3 day Congress will cover topics across the entire spectrum of neuromuscular diseases, including clinical, diagnosis and treatment, as well as controversial sessions and debate on the implementation of new high-cost therapies.

Over the three days there will be 5 plenary sessions and 8 concurrent thematic sessions focusing on diagnostic controversies, thematic updates, standards of care, treatment, implementation of new therapies and more. There will also be 3 thematic workshops in electrophysiology and diagnostics, as well as a poster session of original papers. Also taking place will be up to 7 Pharmaceutical Industry Symposia during breakfast, lunch and at the end of the conference.

In its fourth edition, the SOLANE Congress expects to welcome more than 300 registrants from all over Latin America, not counting the participation of a significant number of national and foreign invited speakers in the field of neuropathies, neuromuscular junction diseases and myology.