The TREAT-NMD Advisory Committee for Therapeutics held its autumn round of review meetings (virtually) throughout November.

TACT hold reviews twice a year to provide expert, multidisciplinary and independent guidance on the translation and development path of therapeutic programmes in rare neuromuscular diseases from either industry or academic groups.

During the autumn meetings we were delighted to welcome four applications. Our bespoke panels of key opinion leaders considered advice on therapeutic programmes on each which included one in Duchenne muscular dystrophy (DMD), one in FSHD and two in myotonic dystrophy (type 1).

The panel will advise each applicant on how they felt they could best proceed with their studies in the form of a confidential report which will be provided to each applicant within 6 weeks of the meeting.

We are also thrilled to be accepting applications for our Spring TACT review meeting which will be an in person event taking place in Philadelphia, USA from the 21st to the 23rd of April 2023. We can accept up to four applications and we are pleased to say we already have a lot of interest for this round. Places are currently still available so if you would like 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.

TREAT-NMD are delighted to announce that the next in our series of free educational events has been awarded ACCME accreditation.

The limb girdle muscular dystrophy (LGMD) masterclass is an online event which will take place on Thursday 9^th & Friday 10^th March 2023.

Aimed at healthcare professionals based in North America, we have a fantastic programme of sessions lined up which will be delivered by key opinion leaders in the LGMD field.  There will be a series of talks, interactive workshops as well as plenty of opportunities to ask questions throughout the two days.

If you are involved in the diagnosis and care of patients with LGMD and would like to learn all the latest updates in the field,  applications are open so apply for your free place today.

 Accreditation and Designation Statement

Psychopharmacology Update Fall 2022

 

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of PeerPoint Medical Education Institute and Treat NMD. PeerPoint Medical Education Institute is accredited by the ACCME to provide continuing medical education for physicians. PeerPoint Medical Education Institute designates the live and enduring formats for this educational activity for a maximum of 14.0 AMA PRA Category 1 Credits™. Physicians should only claim credit commensurate with the extent of their participation in the activity. Live activity dates: March 9, 2023 – March 10, 2023 Enduring activity dates: June 10, 2023 – June 10, 2024”

 

The TREAT-NMD team are doing the final preparations for our 7th International conference which is just around the corner. Taking place from the 7th-9th of December, this is set to be an excellent event with plenty of opportunities to network and some fantastic talks all set on the stunning Vancouver waterfront.

If you’re attending the conference and would like to organise a meeting with one of the TREAT-NMD team members please get in touch at info@treat-nmd.com.  We would be thrilled to meet with you and discuss ways in which we can work together.  You can also stop by our booth to find out more about what TREAT-NMD does and the ways in which we can collaborate. You can view the full conference programme via our dedicated conference website.

If you couldn’t make it this time, you will still be able to follow all the conference action via TREAT-NMD’s twitter and LinkedIn or by searching the #TNMD2022.

CureDuchenne has launched an occupational therapist certification program in order to improve care for individuals with Duchenne muscular dystrophy. The CureDuchenne Occupational Therapist Certification Program provides advanced training to help occupational therapists deliver superior care to meet the specialized needs of individuals with Duchenne as they develop and maintain the skills needed for daily living, working, and independence. The program builds on the organization’s Physical Therapist Certification program, the first program of its kind specifically designed to help individuals living with Duchenne prolong ambulation and delay many areas of disease progression.

Through this program, CureDuchenne Certified Physical and Occupational Therapists are equipped with the knowledge of what stretches, exercises, home program routines and medical equipment are most beneficial, and least harmful, for individuals with Duchenne. These therapists receive ongoing training and assessments, ensuring they are up to date on the most current practices.

For more information, please visit their website or contact Education@CureDuchenne.org.

The French Foundation for Rare Diseases  has created an online course “Diagnosing Rare Diseases: from the Clinic to Research and back”.

This course is designed for individuals with a keen interest in diagnostic research and rare diseases. While primarily designed for medical students and PhD/post-doc students in biomedical sciences, it will also be of interest to Patients Advocacy Organisations’ representatives, Healthcare professionals or paramedics who want to further their knowledge of rare diseases diagnosis.

 

Some key tops covered include:

• The diagnostic process and the types of genetic tests available for rare diseases
• The differences in rare genetic diseases patient pathways
• Technological advances for diagnostic research
• The role of collaborative studies and data sharing in rare diseases diagnosis
• The impact of having a diagnosis or lacking a diagnosis on patients’ lives
• The role and place of physiopathology approaches as well as social sciences research in the context of rare diseases diagnosis.

Learners will gain insight into patients’ experiences, will discuss key issues relating to this topic and will also have the possibility to undertake their own internet-based investigations.

For more information or to enrol click here.

As part of an intuitive to highlight the effort and experience of myologists who are pioneers in their countries, the World Muscle Society (WMS) has conducted a series of interviews with myology experts from around the world.

These insightful interviews dive into the work, motivations and training of these experts which  provides a scientific forum for advances in NMDs’ research and helps promote international collaboration.

The most recent in the series is with  Dr Yuko Shimizu-Motohashi from the National Center of Neurology and Psychiatry, Department of Child Neurology in Japan. Dr Shimizu-Motohashi  discusses her current research/ clinical interest in myology, the most challenging neuromuscular case that she has encountered during her work, regional challenges and more.

You can read the full interview with Dr Shimizu-Motohashi and previous interviews with other experts from around the world here.

As we are nearing the end of the year and looking forward to 2023, here are some key save the dates for interesting events aimed at members of the neuromuscular community:

TREAT-NMD LGMD Virtual USA Masterclass

Date: 9-10 March 2023

Location: Online

UK Neuromuscular Translational Research Conference

Date: 29-30 March 2023

Location: UCL GOSH ICH, London, UK

9th EAN Congress

Date 1-4: July 2023

Location: Budapest, Hungary.

ICN2023: 20th International Congress of Neuropathology

Date: 13-16 September 2023

Location: Langenbeck-Virchow-Haus Luisenstraße 58/59 10117 Berlin

WMS 28: Charleston

Date: 3-7 October 2023

Location: Charleston, USA

Click here to keep up to date with upcoming neuromuscular events and if you have an event you’d like to submit please fill in an event form.

The Myotubular Trust (Registered Charity No: 1137177) was set up in 2006 to raise money for research to find a cure or 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.

In particular, in 2022 the trust would like to encourage research into the involvement of liver abnormalities in myotubular myopathy.

The Myotubular Trust are interested in project grant applications (of 2-3 years’ duration) that could address some, or all, of the following:

• The generation of a greater understanding of the natural history of liver abnormalities in the condition
• Increased insight into the mechanism of action in the liver and bile transport functions in myotubular myopathy
• The interplay between myotubularin deficiency, treatment and liver dysfunction
• The discovery of quantifiable biomarkers that can be predictive or diagnostic of liver involvement, and measured over time for progression / response to intervention

For all proposals, applicants are expected to address how the outcomes of their research will increase the chances of clinical trials moving through to successful outcomes.

The Trust is especially interested in the application of novel approaches and technologies to research into myotubular and centronuclear myopathy. Therefore, collaborations between different medical disciplines and / or different research institutions is encouraged.  They are willing to support research in the UK and internationally if applicants can sign up to  Terms and Conditions and they are also willing to consider applications which involve joint funding of new projects with other organisations.

The Trust may be willing to fund the continuation of a research project (but not to fund an existing project that is already fully funded by another body). Please use the project grant form to apply for such a continuation. If in doubt, please clarify details of any other co-funders and contact them before applying.

The deadline for submission applications is 17:00 GMT on Friday 27th January 2023. Applications will not be accepted after the deadline, but can be submitted at any time prior.

The Myotubular Trust Scientific Advisory Board (SAB), is chaired by Professor Francesco Muntoni of The Institute of Child Health, University College London. The SAB makes recommendations to the Myotubular Trust Trustees on which projects to fund based on scientific merit and peer review.

Further information

Further guidance and an application form can be found here.   If you wish to learn more about the Myotubular Trust, please see their website  or email research@myotubulartrust.org

EURORDIS-Rare Diseases Europe recently celebrated their 25th anniversary by creating some videos to highlight the major achievements and successes over the past quarter-century.

Over the past 25 years EURORIDS have hosted 11 conferences on rare diseases and orphan products, had 15 rare disease days, built up a community of over 1000 member organisations and more.

EURORDIS is a unique, non-profit alliance of over 1000 rare disease patient organisations from 74 countries that work together to improve the lives of over 300 million people living with a rare disease globally.  

You can view their 25th anniversary videos here and to find out more about what EURODIS do and how to get involved please visit their website.

Taking place at UCL Gosh ICH, London from the 29th and 30th March 2023 the UK Neuromuscular Translational Research Conference is aimed at basic scientists and clinical scientists involved in neuromuscular disease research.

The conference is designed as a scientific meeting to promote and share cutting edge clinical and scientific advances in the neuromuscular field, and to encourage new research and collaborations. The event will include invited guest lectures with Q&A, research posters, and flash presentations from early career scientists.

Registration is due to open soon and the abstract deadline is coming up on 10^th January 2023.

For more information you can visit their website