TREAT-NMD Alliance Membership Update

The Summer School of Myology of Paris turns Virtual and Becomes ACADEMYO

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Given the current circumstances, the myology course traditionally held at the Institut de Myologie in Paris, France will now be run virtually this year. The course, renamed ACADEMYO, will be a mix of forty pre-recorded lectures and three live sessions scheduled on 5th, 7th and 9th July 2021.

A platform will be operational for registration soon. Applicants should send their CV to Andoni Urtizberea for prior approval and more information if required.

To maintain an optimal interactivity, the number of applicants has been limited to  100. Priority will be given to new applicants but alumni and industry employees are also welcome.

More information can be found on the Institute's website. 

Duchenne Ireland Funding Research Grants 2021

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The aim of Duchenne Ireland is to raise awareness of DMD at the local, national and government levels. They also aim to raise funds which will go directly to researchers and clinicians who have the best chance of developing improved therapies which will benefit the DMD community. 

Duchenne Ireland is encouraging DMD clinicians and researchers to apply for one of its research grants. Duchenne Ireland's grant funding will ensure DMD translational research moves from the lab to the clinic within a defined timeline. The deadline for applications is 31st May 2021. 

If you'd like to learn more about the funding available please contact Duchenne Ireland.

European Alliance for Newborn Screening for SMA Publishes First White Paper

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The recently formed European Alliance for Newborn Screening for Spinal Muscular Atrophy, composed of European patient organisations, academics and the pharmaceutical industry, today published its first whitepaper, entitled "Spinal muscular atrophy: screen at birth, save lives", wherein it calls upon all European states to introduce routine screening of all newborns for spinal muscular atrophy (SMA) by 2025.

SMA therapies, when provided before symptom onset, can significantly reduce, and may prevent, disease manifestations. 

However, only routine newborn screening for SMA makes it possible to diagnose the disorder at its earliest stage and immediately introduce disease modifying therapies that reduce and may potentially prevent disease symptoms. Screening at birth also permits to introduce the best standard of medical care, whilst parents and children no longer have to go through the pain of diagnosis when they learn about facing a life with severe disabilities.

The Alliance seeks to help foster the introduction of universal newborn screening for SMA in Europe and inform a systematic dialogue within European healthcare systems, with an involvement of healthcare experts, policymakers and patient advocates.

The Alliance calls on all European countries to ensure that, by 2025, all children in Europe are screened at birth for 5q SMA due to SMN1 gene mutation. 

For more information please contact the Alliances' press office at nbsalliance@sma-europe.eu or +34 653 700 739

TREAT-NMD Services Limited is Looking for a New CEO

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TREAT-NMD Services Limited are looking for a new Chief Executive Officer. 
The ideal person will have previous operational management experience, a proven track record for effective team management and results-driven leadership, preferably from working within an small and medium sized enterprises, Charitable/3rd Sector organization or Health Care organization.

You will have a proven ability to effectively manage a growing SME, understand corporate responsibility, be able to liaise with stakeholders from a variety of academic and health care settings internationally, and implement systems and processes to support sustainable growth.

Previous experience in the role of CEO is advantageous, although those with experience in a Chief Operational or Managing Director role, with a desire for a new challenge, are also encouraged to apply.

The CEO will work closely with the TREAT-NMD Global Alliance Executive Committee and the Board of Directors of TREAT-NMD Services Ltd, serving as the director of operations and acting as the main link between the TREAT-NMD Global Alliance and the service functions delivered by the different divisions within the company.

More information on the role can be found here. The closing date for applications is 10th May 2021.

TREAT-NMD Global Registries Platform

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It is a busy time for the TREAT-NMD Global Registries Platform project as we move into the next phases of the project before the launch at the end of May. The build of the patient portal is now moving into the beta testing phase, where we have several testers engaged to test and provide feedback. The platform team is currently engaging with TGDOC Member Registries to discuss the implementation of the platform and the datasets. This process is also helping the platform team to understand how each registry may plan to use the platform after the launch date.

Registries using the platform will be identified as one of the following:

• Option 1 Registry, which are registries that wish to use the platform as their main data repository, and where data transfers to the Central Data Warehouse are completed within the platform,
• Option 2 Registry is a registry that intends to remain with their current/existing platform and will be able to provide data via secure transfer to the Central Data Warehouse using a data transfer toolkit.

While we encourage and support all TGDOC Member Registries to share data via the platform, we acknowledge that this may not be possible for all registries or might take some time to implement. These registries will of course continue to participate in our usual TGDOC Registry Enquiries. As we progress with our roll out we will be working closely with these registries to understand their challenges or reasons for not participating via the platform to determine how we can best work together to safely and securely capture and share data, in a unified and standardised manner that allows for efficient and effective high quality data analysis and reporting.

The LGMD module build is also progressing with the clinician portal almost complete and will soon be ready for the feasibility study phase of the dataset project. The patient portal is also nearing the end of its development phase and will soon be ready to be built within the platform software, where we will be alpha testing during its update releases.

We are continuing to update our stakeholders for the project, but if you would like to be involved in testing, offer support or advice in relation to the platform or would like to see a demonstration of the platform please feel free to contact Caroline directly using the email below or the link on our webpage. We are keen to engage with as many people as possible.

For further information on the TREAT-NMD Global Registries Platform or to participate as a ‘test registry’ then please contact Caroline Ogden, Project Manager or visit our website at to learn more.

19th Asian-Oceanian Myology Center Meeting 2021

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AOMC2021, the 19th Asian-Oceanian Myology Center Meeting, will be a hybrid event taking place both virtually and on-site at BEXCO, Busan, South Korea from 10th-11th June. 
The scientific programme will cover the most important fields of neuromuscular disease including clinical characteristics, pathophysiologym and innovative diagnostic technology and treatments of NMDs. Clinical-pathological conference, which is the most traditional and symbolic program of AOMC will be played on a platform that is fully interactive between Chairs, speakers and participants. 

More information about the conference, registration and how to submit a poster abstract can be found here. 

EURO-NMD Survey on Educational Needs in Neuromuscular Diseases

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The European Reference Network (ERN) on Rare Neuromuscular Diseases (EURO-NMD) is aiming at remodelling its training offers and wants to hear from the community it serves.

ERNs are continuously working to improve the training and educational offers implemented by the networks to overcome the gap in the existing curricula for rare diseases training. This survey is aimed at identifying the emerging training needs among EURO-NMD healthcare professionals, with the objective of establishing the network’s educational priorities for the years to come, as well as the appropriate means and tools to address them. The ambition is to structure a European postgraduate training curriculum on rare Neuromuscular Diseases for a new generation of highly skilled physicians, other healthcare professionals and scientists.