Newsletter - April 2019 ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏
Newsletter - April 2019

Early Bird Reigstration Fee for Summer School is Extended!

There is still time to apply to attend our Translational Research Summer School!

This course which has been especially developed for researchers and clinicians interested in translational research is in association with TREAT-NMD. The week-long course addresses the following aspects as it travels along this established pathway.

  • Bench to bedside research
  • Regulatory system
  • Clinical trials
  • Outcome measures
  • Patient communication
  • Registries and biobanks
  • Biomarkers and –omics

1st-5th July 2019 - Leiden, Netherlands

Attendees of the summer school will discover how networks such as the European Reference Network EURO-NMD and TREAT-NMD work and collaborate with patients and regulators to facilitate therapy development.

To ensure a high quality experience for all attendees course numbers are kept low and we recommend you register promptly to reserve your place at the very best price of €400 (for regular attendees).

Early bird registration will close on Friday 10th May!


Meeting Report from Myology 2019

  Meeting Report from Myology 2019


Myology 2019, was the sixth edition of the International Congress of Myology organized by AFM-Telethon, and took place in Bordeaux, France, from March 25 to 28.

More than 800 researchers and scientific experts from 35 countries worldwide gathered for the congress. This year's congress consisted of seven plenary sessions, 12 parallel sessions, 5 industry symposiums. Over 400 scientific posters highlighted current issues related to myology, discoveries and new developments, progress in the identification of new genes, and innovative therapies including the latest developments in gene therapy for the treatment of the entire muscle mass.

The congress showed that we are entering in a new era for neuromuscular diseases, namely the era of therapeutics, whether they be innovative therapies derived from genetics or molecule repositioning. The hallmark of this congress was that it gathered both fundamental research and applied research with very talented and determined experts from around the globe. With new molecules, new mechanisms, new concepts, new approaches, new trials, new treatments and new hopes for pathologies which, until that time, had no prospect. Hope becomes a reality.

EURO-NMD at Myology 2019

EURO-NMD helpdesk assistant Chloe attended on behalf of the ERN. A number of congress delegates who are affiliated with our network took this opportunity find out more about the Clinical Patient Management System, ask any questions they may have and receive a little one-to-one tuition on the system.

Overview video

Find out more about this year's congress from a specially made overview video in which Serge Braun discusses how beneficial this congress has been at this very exciting time within therapy development.


EURO-NMD at European Academy of Neurology in Oslo

  Myology 2019

We are delighted to announce that representatives of EURO-NMD will be at the European Academy of Neurology in Oslo, Norway 29th June - 2nd July.

Throughout the four days of the congress EURO-NMD will have an information booth and we would love it if you would drop by to say 'Hi' and find out a little more about our network and how it benefits the neuromuscular community.

For those of you affiliated with the network and would like to find out about Clinical Patient Management System (CPMS) our helpdesk staff will be on hand to help out. You will be able to see the CPMS in action, have your questions addressed and receive one-to-one help with the different aspects of the system. To arrange a meeting to look at the CPMS whilst you are in Norway then please contact Chloe Blewitt or alternatively 'call in' to the booth when you're passing!


Experts meet to discuss FAIR data for Duchenne

FAIR Data WDO March 2019  

The World Duchenne Organisation (WDO) and several of its members have been advocating for better use of Duchenne data, which is currently kept in silos.

WDO does this by bringing this subject to the attention of the EU lobby, by developing tools enabling better data sharing, such as the Duchenne Data platform, and organizing meetings on how to enhance data sharing for Duchenne. On March 21 and 22, 2019 during a meeting the FAIR initiative, the Personal Health Train and next steps related to data visiting were discussed at our meeting in Amsterdam attended by representatives of registries, patient advocate leaders, clinicians, scientists, companies and regulators.

WDO are currently planning a manifesto, a 'Bring your own data' workshop and extensive meeting report. We recommend that anyone interested in this area not only visit the WDO website but also sign up to the WDO newsletter to ensure they find out about any developments in this exciting area.


10 reasons to join Share4Rare

Share4Rare's new video  

We increasingly use the internet as a resource for health information. We also use social media platforms to connect with others living with similar conditions. However, we cannot always be certain that what we are reading is from a trustworthy source or that social media platforms are protecting our privacy.

Share4Rare is a new digital platform that aims to address the needs of patients, families and researchers. The platform will provide accurate information about rare diseases and provide a safe space to interact and share information.

Why should you join Share4Rare if you are a patient or a relative?

  1. S4R is a European Commission funded project under the leadership of the coordination team at Hospital Sant Joan de Déu Barcelona.
  2. Patients and patient advocates have participated in the design and development of the platform through co-design workshops.
  3. The platform connects users across diseases and geographic locations.
  4. The platform guarantees confidentiality.
  5. S4R has the approval of the Ethics and Research Committee of Fundació Sant Joan de Déu.
  6. Only authenticated users can participate in the platform.
  7. The S4R patient advocacy toolkit empowers patients to participate in, drive and lead research initiatives.
  8. Access to accurate, up to date medical content.
  9. Participate in contributing data to help develop new research.
  10. Promotion of research initiatives based on clinical data donation.
The full article on the benefits of joining S4R is now available, alternatively find out more about the project here.

You can also find out more about this unique and innovative project from their latest video which has recently been released.


EURO-NMD participates with Guideline Production Group of the European Academy of  Neurology

The Guideline Production Group of the European Academy of Neurology aims to prepare a guidance for developing and reporting guidelines in the field of rare neurological diseases. 

To achieve this, they are organising a Delphi consensus process among several experts in this field. The scope of the consensus is to define methodological problems peculiar to guidelines for rare neurological diseases, and to collect and rate suggestions on specific issues for their development and reporting.

EURO-NMD's coordinator Teresinha Evangleista has been selected as a potential participant within the core-group that will be involved in the initial idea generation and will participate throughout the whole process which will culminate in a final paper which will be published in the European Journal of Neurology in due course. 

We will of course, keep you informed with progress updates and also will highlight the article once published.


Next EURO-NMD Webinar

The next EURO-NMD webinar will take place on...

Thursday 7th May at 11:00am (CET)

The webinar will be presented by Kasper Bedsted (DG SANTE) and will be a CPMS training webinar to show the different features of the platform and how to submit a panel.

Details of how you can participate in the webinar can be found in the webinar area of our website. We hope you can all attend!

If you have any suggestions for our upcoming webinars please contact Chloe Blewitt.

Save the Dates for the Upcoming EURO-NMD Board Meetings!

Two dates for those of you who are involved with EURO-NMD are...

19th July 2019 in Paris, France
6th November 2019 in Ferrara, Italy

Save the dates and we look forward to seeing you there!



Our Clinical Patient Management System currently has 37 active users working on a number of live panels.

The main focus at the moment is to continue creating panels and for users to begin accessing the system on a regular basis to familiarise themselves with the system and contribute to panels.

Following the recent IT Advisory Group Meeting in Brussels we identified some difficulties with login process and the layout of the panels within the CPMS itself.

DG SANTE and the Openapp developers took our comments on board and are looking to address these in the coming version releases of the system.

As always, if you have any questions, comments or would like some advice about the CPMS please do get in touch via our helpdesk or by email.