2022 TREAT-NMD Conference

Virtual Booths Available for the Myotonic Dystrophy Foundation 2021 Conference

‌

The Myotonic Dystrophy Foundation 2021 Virtual Conference will be taking place on 10th-11th September. The event will featured educational and community building programming specifically for the DM community. For  information about the programme, speakers and more you can visit the conference website. 

Like last year, the conference will be held completely online, and will offer the unique opportunity to connect with attendees through a customised exhibitor booth. The booth is highly interactive and can feature content including photos, videos, polls and more.The conference platform will also provide an opportunity to connect with attendees 1:1 before, during and after the Exhibitor & Sponsor Showcase. 

If you’re interested in hosting a booth, please contact the Myotonic Dystrophy Foundation. 

Romanian Translation of the Duchenne Muscular Dystrophy Guide for Families Now Available.

‌

We are excited to announce that a new addition to our collection of Family Guide translations is now available.

Due to the fantastic hard work of the Parent Project MD Romania we now have the Standards of Care Family Guide available in Romanian.   

This latest translation means that our Family Guides are now available 14 different languages.  These fantastic resources are free to view on our website and we will continue to add more translations in the next few months. 

If you would like to find out about printing any of the guides or require further information, please contact the education team.

Coalition to Cure Calpain 3 Issues Request for Applications for Research Related to LGMD2A/R1

‌

Coalition to Cure Calpain 3 requests applications for research and translational projects related to calpain 3 and limb-girdle muscular dystrophy type 2A (LGMD2A, also called LGMDR1 Calpain 3-related). The goal of the RFA is to enable the development of therapeutic approaches for this disease. This opportunity is open to investigators from both US and non-US based academic and biopharmaceutical organizations. Letters of Intent are due by September 1, 2021, with full applications due by November 1, 2021.

The link to the RFA can be found here. 

Can you Help Translate the Care Questionnaire Study for LGMD

‌

On behalf of the TREAT-NMD LGMD advisory group, two surveys have been developed with the goal of understanding current care practices around the globe to help shape eventual Standards of Care for those living with LGMD.  We are currently reaching out for volunteers to assist with additional translations of the patient and clinic surveys to enable as many responses as possible. We would also encourage advertisement and dissemination of the questionnaires within your own patient networks, registries, and clinics.

The patient survey aims to give patients, family, and caregivers the opportunity to report their clinical care experiences.  This survey is currently available in English, Spanish, German, Dutch, Portuguese, French, Korean, Norwegian, Russian, Japanese, and Ukranian with Italian, Urdu, and Afrikaans pending. If you or someone you know would be interested in providing translation for another language, please reach out to Lindsay Alfano.

Similarly, the clinic survey is aimed at clinicians that diagnose and/or manage the care of persons with LGMD. It is currently translated into English, Portuguese, Japanese, Russian, and Spanish languages. If you have a suggestion for another useful translation or are able to volunteer to translate this survey, again, please contact Lindsay Alfano. 

TREAT-NMD LGMD Latin American Masterclass - Have you booked your place?

‌

The TREAT-NMD LGMD Latin American Masterclass is a free, educational online event for healthcare professionals, which will be taking place on the 3rd and 4th November 2021.  The course will be delivered in both Spanish and Portuguese languages and there is still time to reserve your place. 

For more information about the Masterclasses offered by TREAT-NMD, or to apply for your place, please visit the TREAT-NMD website. 

Nominations Now Open for the EURORDIS Black Pearl Awards

‌

The eleventh edition of the EURORDIS Black Pearl Awards will be taking place online on 8th February 2022. The awards bring together persons living with rare disease, patient organisations, policy makers, scientists, healthcare professionals, industry representatives and more to acknowledge the achievements and exceptional work of people making a difference for the rare disease community.

Now is your opportunity to nominate a star of the rare disease community for one of the awards. The 12 award categories recognise the outstanding efforts of individuals, organisations, companies, researchers, scientists, media, and policy makers in bringing about change to improve the lives of the 30 million people in Europe and 300 million worldwide living with a rare disease. The deadline for nominations is 10th September 2021. 

If you'd like to learn more, submit a nomination or watch the 2021 awards ceremony please go to the Black Pearl website. 

TREAT-NMD Education Committee Seeks New Member

‌

After three years, valued member of the TREAT-NMD Education Committee, Elizabeth Vroom is stepping down.  The TREAT-NMD Education Committee (TEC) is a board of experts who have overall responsibility for overseeing the quality of all educational materials and events created by, linked to or endorsed by TREAT-NMD.  We would like to thank Elizabeth for all her contributions to the committee over the years, her experience and guidance has been extremely valuable in developing a variety of educational programmes and events in the Neuromuscular field. 

Although we are sad to say goodbye to Elizabeth we now have an exciting opportunity to find a new member for the committee, if you would like to apply please complete the Application Form to be considered.

WHO Review of the Essential Medicines List for children (EMLc)

‌

The World Health Organisation (WHO) is initiating a comprehensive analysis of the most needed medicines for children to inform the review of the Essential Medicines List for Children (EMLc). 
Global Accelerator for Paediatric formulations (GAP-f), in collaboration with Penta, is performing a survey to collect the health care professional experience with medicines for children as the formulations identified as most problematic and why, and which medicines are missing or are not accessible for the children and why. 

The survey should take no longer than 15 minutes to complete and responses should be submitted by 31st August. 

FSHD Society Masterclass Announced

‌

The FSHD Society has designed a state of the art online CME course for clinicians involved with the diagnosis and management of care for patients with FSHD will be taking place on 12th August (12pm-5pm Eastern Time). 

The course will present information on the latest in diagnosis, genetic counselling and testing, natural history, symptom management, and standards of care. This course will also provide 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. 

To register and for more information please go the event website. 

World Muscle Society Website Expands Community Content

‌

The World Muscle Society (WMS) has been working on developing its website. These changes include some new and broader content for the neuromuscular community. One of the new sections is focussed on interviews which more recently include a conversation with the winner of the prestigious Presidents Prize for Young Miologist of the Year from the WMS Congress in 2020,  Payam Mohassel. 

Payam is a clinical research fellow at the NIH. After his clinical training in adult neurology at Johns Hopkins University, he joined Dr. Carsten Bönnemann’s group at the NIH. His main areas of research interest include gene discovery efforts, disease mechanism studies in preclinical models, and early-phase clinical trials in neuromuscular disease.

From its inception, the core belief of the WMS has been to nurture the young and to support and encourage them from embryonic myologist to full maturity as scientists and practitioners, establishing and growing their own teams of enthusiastic young researchers.

If you would like to read the full interview, or find out more about the 2021 WMS Congress, you can visit the WMS website.  

Registration Now Open for Third International Conference on Imaging in NMD 2021

‌

Registration and abstract submission are now open for the third International Conference on Imaging in Neuromuscular Disease. The conference will take place on the 8th and 9th November 2021 as a live online event. This year’s conference will be chaired by Pierre Carlier and Hermien Kan and features internationally recognised speakers including Melissa Hooijmans, Werner Stenzel, Dominik Weidlich and Andoni Urtizberea.

The conference programme is spread across two days and features lectures, debates and poster presentations with awards. Further details about the programme can be found on the website.

Young researchers and trainees are encouraged to attend and participate in the conference. Abstracts will be featured as presentations prior to the live event on the conference app. Delegates will have the opportunity to post comments and questions, then poster pitches will be presented during the live event. The deadline for abstract pitches is 1st September 2021. Further details regarding submission can be found here.

The third International Conference on Imaging in Neuromuscular Disease is endorsed by the International Society for Magnetic Resonance in Medicine and the European Reference Network NMD.

For sponsorship opportunities contact Clare Bradley

Myotubular Trust Launches 2021 Grant Call

‌

The Myotubular Trust has announced their 2021 call for research grants and there is still time to apply. The trust are looking to fund projects that will help find a cure and/or a treatment for any form of centronuclear and myotubular myopathy (congenital X-linked recessive; congenital autosomal recessive; autosomal dominant), focusing on research that would not generally be funded by public or industrial funding sources. 

Applications may be made for: 

1. Project Grants - the Trust will consider applications from the Principal Investigator for projects 2-3 years duration to be carried out by a Post-Doctoral researcher or PhD student. 

2. A Myotubular Fellowship, non-clinical - the Fellowship scheme is designed to support early career scientists to help them develop their independence. 

Completed applications should be submitted by 5pm GMT on Friday 29th October 2021. The Trust anticipates that awards will be made in April 2022. For more information please visit the Myotubular Trust website. 

VCP Scientific Conference Goes Online

‌

Academics, researchers, industry, government agencies, and healthcare professionals are invited to attend the upcoming VCP Scientific Conference taking place virtually on September 9-10, 2021. The conference will bring together lab scientists, clinician-scientists, industry researchers, and patient leaders to discuss topics related to the Valosin Containing Protein (VCP). The multidisciplinary nature of the conference will significantly impact the innovation and coordination of the research and accelerate translational discoveries in the field to bring about therapeutics in the near future.

All are invited to submit a poster and/or presentation abstract for the 2021 VCP Scientific Conference. Prizes will be awarded for the top three posters. To find out more about submitting a poster please visit the conference website. Learn more at