Double TREAT-NMD Expert Masterclass Success

EURO-NMD Survey on Female Carriers of Dystrophinopathy Needs You!

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With the aim of organising the 263rd ENMC workshop “Focus on female carriers of dystrophinopathy: refining recommendations for prevention, diagnosis, surveillance and treatment” EURO-NMD would like to ask for your collaboration regarding the care provision for Duchenne muscular dystrophy (DMD)/Becker Muscular dystrophy (BMD) carrier females at your Institution. If you're a clinician and would like to participate please complete a short survey, which can be found here.

The deadline to complete the survey is 31st May 2021 and your answers will provide EURO-NMD with a better understanding on the current situation across Europe and will inform the development of recommendations. 

TREAT-NMD Advisory Committee for Therapeutics (TACT) Hosts 23rd Meeting

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The TREAT-NMD Advisory Committee for Therapeutics held its 23rd meeting (virtually) 30th April to 1st May. TACT meets twice a year to provide expert, multidisciplinary and independent guidance on the translation and development path of therapeutic programs in rare neuromuscular diseases from either industry or academic groups. 

The spring meeting considered two applications for advice, on this occasion, both in Duchenne Muscular Dystrophy. We were delighted to welcome an industry application from Pharmaxis; and an academic group - Dirk Fischer from the University of Basel with support from Emily Crossley at Duchenne UK. 

The TACT core committee and secretariat put together a bespoke panel of experts to advise each applicant on how to proceed with their studies and a report will be provided within 6 weeks of the meeting. 

We are hopeful that the next two meetings, Philadelphia on 23-24th of October 2021 and Europe (location to be confirmed) on 23-24th April 2022 will be in-person events. You can read more about TACT by going to the TREAT-NMD website. 

For more information on the TACT review process or to submit or discuss an application for advice, please contact the TACT Coordinator, Cathy Turner, or TACT support, Laura Robertson.

EURO-NMD and TREAT-NMD Translational Research Summer School in Winter

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EURO-NMD is delighted to announce that applications are now being accepted for the 2021 five day winter school.  Approximately 30 participants will be welcomed to the Leiden University Medical Centre in the Netherlands in December.

The annual course has been developed for researchers and clinicians interested in translational research and includes sessions on the regulatory system, clinical trials, outcome measures and much more. 

The Translational Summer School, Leiden, Netherlands, has been accredited by the European Accreditation Council for Continuing Medical Education (EACCME®) with 26 European CME credits (ECMEC®s).

It should be noted that although EURO-NMD anticipates that the face to face school will go ahead, delegates should be mindful that travel for attendees and speakers might not be possible. There is still a possibility that this school may have to be held online.

More information on the summer school can be found on the EURO-NMD website.

TREAT-NMD Education Committee (TEC) Endorsement

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Is your organisation planning an educational event about a neuromuscular disease? Did you know you can 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 seal of approval which can be awarded to a range of educational programmes including live events, e-learning courses and blended learning sessions. The endorsement demonstrates that your education programme meets a high standard and provides assurance to your attendees about the calibre of your course.

All applications are reviewed by TEC, a board of experts (academics and patient representatives) who are affiliated with TREAT-NMD and have an interest in the educational 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 on our website and you can also apply for endorsement on this page. Please email: education@treat-nmd.com  if you have any questions or would like further information about the endorsement process.  

Updates to World Muscle Society (WMS) Website

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WMS has been working to update its website and would like to highlight some of their new pages which may be of interest. 

Do you need to promote a new job vacancy in the field of neuromuscular science? WMS may be able to help - it has a new Job Posting website page. To advertise your post all you need to do is enter your details into the form template and click submit. Submitted vacancies will then be reviewed by the WMS committee prior to listing.

For non WMS members, they will be directed to create a WMS website account so they can submit the form. So a colleague (not the WMS member) for example can submit the post. 

Do you need to advertise an event? Do you have a relevant conference, workshop or event you'd like to advertise to WMS members and followers? The WMS website now has a new Events Page on the website, so WMS can feature and promote your event on your behalf. Visit the events pages of the WMS website and simply complete the 'Submit Form'. Please note all events are subject to review prior to listing. 

If you have any queries, please contact Clare Beach, WMS Secretariat. 

World Duchenne Awareness Day 2021 Theme Announced

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World Duchenne Awareness Day takes place on 7th September. On this date the World Duchenne Organisation raises awareness of Duchenne and Becker Muscular Dystrophies around the world. Each year has a theme and for 2021 the theme will be 'Adult Life & Duchenne'.  Over the past decades life expectancy of individuals affected by Duchenne MD has increased significantly and adults with Duchenne are not an exception anymore. Adult life with Duchenne comes with new challenges and opportunities as well as with new medical issues. This World Duchenne Awareness Day will be an opportunitiy to advocate globally for young adults and adults with DMD/BMD so that they may live life to the its fullest. 

On 7th September the World Duchenne Organisation will be hosting an online event where experts share their stories and experiences surrounding BMD/DMD and adult life. With the aim of inspiring and empowering young adults and their families to think and plan for the future. 

If you would like more information about Duchenne Awareness day please visit the World Duchenne Organisation website. 

Survey on Patient Organisations' Knowledge and Position Paper on Screening for Inherited Neuromuscular Diseases in Europe Published

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 A new study aimed at the evaluation of the state of play for the screening of inherited rare neuromuscular diseases in Europe and patient needs has been published in the Orphanet Journal of Rare Diseases. 

The patient advisory board of the European Reference Network for Rare Neuromuscular Diseases (EURO-NMD) conducted a survey on patient organisations' knowledge on screening for inherited neuromuscular diseases in Europe with 30 patient organisations from 18 European countries. This study revealed that most patient organisations were in favour of systematic screening with the option to opt-out and identified early access to treatment as the main priority to perform screening for NMDs. This study highlighted the need for international coordination leading to a common policy which would harmonise the screening for inherited neuromuscular diseases throughout Europe. 

Programme Published for ACADEMYO

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ACADEMYO, formerly the Summer School of Myology of Paris, will be hosting a myology teaching course from 5th-7th-9th July. 

The tentative programme for the event has now been published and will offer attendees the opportunity to view both live and pre-recorded events. The lectures on demand will be available from Mid-June until the end of July and will include talks on topics such as muscle anatomy and physiology, muscle imaging, outcome measures in NMD and much more.

Registration fees are 300 euros for academic applicants and 1,000 for applicants from industry. There is still time to register and potential applicants can do so by emailing their CV to Andoni Urtizberea. For more information please go to the website.