The 2019 Neuromuscular Translational Summer School will be taking place from 1st to 5th July in Leiden University Medical Center in the Netherlands.

The aim of the school is to facilitate the clinical development of therapies for NMDs , to educate clinicians and researchers working in the NMD field on aspects relevant for translational therapy development and to outline and showcase how networks like EURO-NMD and TREAT-NMD facilitate therapy development

The EURO-NMD summer school, which is developed in association with TREAT-NMD, is specifically developed for researchers and clinicians interested in translational research. The week-long course addresses the following aspects as attendees move along an established pathway.

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

Attendees also have the opportunity to find out about the various interactions that take place between key stakeholders, how the regulatory system works, including and being involved in indepth discussion on ethical issues in clinical trials. There is also an opportunity to take part in a TACT (TREAT-NMD Advisory Committee for Therapeutics) style review.

There is also the chance to find out about various outcome measures and gain an appreciation of the need for standardisation and training in this area. Participation in a clinical trial practicality forum as well as a patient communication workshop which focusses on patient communication also forms part of the curriculum.

Attendees who complete this course will gain the knowledge of the many different aspects that are involved in translational research and also have a deeper understanding and compassion for those involved in the whole trial process from researcher to patient.

If you would like to register your interest in the event you can do so via the EURO-NMD website.

Two million pounds (UK£) are available to fund the best research proposals into the treatment of Duchenne muscular dystrophy.

Duchenne UK, a patient organisation based in the UK with global reach, is pleased to announce the launch of their 2019 Grant Call. They wish to invite research proposals for submission and review.

Specifically, they are interested in receiving proposals relating to two therapeutic approaches:

i) Treatment of the root cause of the disease through innovative gene therapies and/or more effective therapy delivery methods

ii) Novel approaches designed to regenerate muscle fibres and/or remove/reduce fibrosis in muscles

This call has a broad remit in terms of drug development stage. Proposals that are in early preclinical stage, but which show great promise as alternative approaches are welcomed, as are drug development projects with the potential to be in clinical trials now or in 2–4 years and which have compelling pre-clinical data in appropriate animal models.

How to make your submission?

In the first instance send an email to Dr David Bull and Megan Mullany ([email protected] and [email protected]) indicating which of the two therapeutic approaches you are interested in, a brief (500 word) outline of the project and approximate costs. If you meet the criteria, Duchenne UK will then send you a detailed submission template which you should complete in full and return.

Full submissions, sent on the template referenced above, should be received at the latest by: Monday 18th March 2019

Co-founders of Duchenne UK, Emily Crossley and Alex Johnson said:

"Supporting and accelerating innovative research is at the heart of what we do at Duchenne UK and we are pleased to be offering these grants of up to £2million. Gene therapy is offering great promise, but challenges exist, especially in areas like antibody status. We hope that this call will encourage groups working in these areas to submit applications and engage with us on bringing new treatments to the clinic.”

More information about the call and the review of submissions can be found here: https://www.duchenneuk.org/news/grant-call-2019

We are thrilled to announce that TREAT-NMD is now accepting applications for the 5^th masterclass in Duchenne muscular dystrophy (DMD). This will take place at the Movenpick Hotel Amsterdam City Centre, the Netherlands, on Thursday 16^th and Friday 17^th May 2019.

The masterclass is suitable for health care professionals and researchers who are involved in DMD care and research (both paediatric and adult). The draft programme can be seen here: https://www.treat-nmd.eu/events/752/

If you would like to apply for a place, please complete the application form: https://www.treat-nmd.eu/events/752/.

Travel and accommodation will be covered and places are highly competitive so it is important that this workshop will have significant impact on your work. Places will be allocated based on the widest benefit to the DMD patient community and will be decided on by the TREAT-NMD Education Committee (TEC). You will be asked to explain how the Masterclass will benefit your work as part of the application process.

If you have any questions please contact the DMD Liaison Officer Cathy Turner at the John Walton Muscular Dystrophy Research Centre, Newcastle University, UK: [email protected]

Closing date for applications is 28^th February 2019

The Myotubular Trust (UK Registered Charity No: 1137177) was set up in 2006 to raise money for research to find a cure and / or treatment for any of the genetic forms of centronuclear and myotubular myopathy.

There are a number of genetically distinct forms of centronuclear myopathy. The most common form is x-linked, usually called myotubular myopathy, affecting in the vast majority of cases, and only boys. It is also usually the most severe, presenting in the new-born period with associated breathing and swallowing difficulties in addition to the general muscle weakness. The other forms are either dominant or recessive in inheritance, are usually, but not always, milder, and vary widely.

2019 call for projects

Myotubular Trust is holding a 2019 call for research grants. We will require completed applications by 17:00 (GMT) Friday 15^th March 2019. We anticipate making awards in late June / early July.

We are looking to fund further 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. This call will be open to research bodies internationally.

We will be looking for the following types of application:

1. A project grant applied for by a Principal Investigator to fund a project for 2-3 years duration to be carried out by a Post-Doctoral researcher, or PHD student

2. A Myotubular Trust fellowship – basic science (3-4 years duration), where the scientist has identified a group that he or she wants to work with. Award is made to a named individual.

In particular, we would like to encourage the application of new technologies to research into centronuclear and myotubular myopathy; interventional trials; and those which may involve collaboration between different medical disciplines and / or different research institutions. We are also willing to consider applications which involve joint funding with other organisations.

Myotubular Trust’s 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 assessment and peer review.

Further information

Further guidance and application forms can be found on the website - https://myotubulartrust.org/research/grants-process/

If you wish to learn more about the Myotubular Trust, please see our website http://www.myotubulartrust.org or email [email protected]

The John Walton Muscular Dystrophy Research Centre are seeking two Clinical Research Fellows. This is an exciting opportunity to join on of the world leading centres for Neuromuscular research.

All posts benefit from strong international collaborations and provide opportunities to contribute to scientific conferences, publications and audit projects.

Responsibilities include:

• Patient Recruitment
• Clinical Assessments
• Participation in Neuromuscular clinics

The following opportunities are available:

• Vision DMD Clinical Research Fellow - Full time post until 31st December 2019 - Closing date 15th February 2019
• Unite DMD Clinical Research Fellow - Part time post (50%) for up to 4 years - Closing date 15th February 2019

Key Requirements

The candidates must have a degree in medicine or equivalent and must have a full GMC licence to practice. Candidates should have experience of and commitment to multidisciplinary working. The candidate is expected to have proven ability to organise and prioritise workload, work and communicate effectively within a team environment and use own initiative.

For informal enquiries please contact Becky Crow.

The Second International Conference on Imaging in Neuromuscular Disease will be held at the Langenbeck-Virchow-Haus in Berlin from 17th to 19th November 2019.

The conference will feature internationally recognized speakers including Pierre Carlier, Andrew Blamire, Pierre Yves Baudin, Julien Valette, Daniel Ennis, Olivier Scheidegger, Bram van Ginneken, Jennifer Kollmer and Peter Caravan. The programme will include sessions on new imaging applications in NMD, diffusion imaging, imaging cardiac muscle, deep learning and MR neurography.

Young researchers and trainees are encouraged to attend and participate. Selected abstracts will be featured for platform presentation during the sessions and all posters are eligible for poster awards.

Registration for this conference will open soon. We look forward to welcoming you to Berlin in November 2019. For sponsorship opportunities please contact Helen Sutherland.

This conference has been endorsed by the International Society for Magnetic Resonance in Medicine.

TACT, the TREAT-NMD Advisory Committee for Therapeutics, evaluated three proposals at its second 2018 review meeting, which took place between 3rd – 4th November in Chicago, USA. The full TACT reports with recommendations were sent to the applicants within 6 weeks of the meeting and the corresponding non-confidential summaries, approved by the applicants, will be uploaded to the TACT webpage on the TREAT-NMD website in the next few weeks.

The proposals reviewed were:

• Multiple-ascending dose study for SRP-5051 in patients with Duchenne muscular dystrophy, submitted by Dr Dugar, Sarepta Therapeutics, Switzerland
• Use of anti-miR106b inhibitors for promoting muscle regeneration in the treatment of DMD, submitted by Dr Aránega, University of Jaén, Spain
• Clinical development of omigapil in congenital muscular dystrophy CMD, submitted by Dr Coserea, Santhera Pharmaceuticals Ltd, USA

The next TACT review meeting will be held in Glasgow, UK between 12-13 April 2019 and we have received four applications to be reviewed.

If you are interested in submitting an application to the following meeting taking place on 2nd – 3rd November 2019 (location tbc), please contact Cathy Turner at [email protected].

TACT is funded through contributions from industry applicants and by generous support from patient organisations including PPMD, Cure Duchenne, MDUK, MDA, Joining Jack, Duchenne UK, Duchenne Research Fund, Duchenne Ireland, Myotubular Trust, Duchenne Now, Duchenne Children’s Trust and SMA Europe.

For more information about TACT and previous applications, please visit our website.

In 2018, the TREAT-NMD Executive Alliance Committee invited you to become a member of TREAT-NMD. Our membership total rose throughout 2018 but we were short of reaching of 1,000 member target. We are hoping that will change in 2019 and with the new year will come a multitude of new membership applications.

TREAT-NMD membership is free and our indivdual members will benefit from:

• Discount on TREAT-NMD conference registration fees
• 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted
• Opportunity to nominate yourself or nominate a TREAT-NMD member to fill a vacancy on the Executive Committee
• You get to vote on a number of TREAT-NMD matters!

Organisational membership is free and benefits include:

• Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD
• Organisational members will be able to use the TREAT-NMD logo on their website to show that they are affiliated with TREAT-NMD
• 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted

To find out how to become a member and to apply, please click here.

If you are already a member and have recently changed your email address, please notify us of your new address by emailing [email protected].

Interested in submitting an article or event? We invite news (including conference/workshop) summaries, articles and upcoming events concerning all aspects of translational research and for all neuromuscular disorders. To submit an article or event please go to the dedicated page on the TREAT-NMD website and complete the online form. Be sure to include an JPG image and a link to where readers can find further information.

The next edition of the TREAT-NMD Newsletter will be published on 28th February 2019.

Both TREAT-NMD organised events, and those organised by external parties, will be listed below.

Rare Disease Day

28th February 2019

An annual event on the last day of February to raise awareness for rare diseases amongsth the general public, healthcare professionals and decision-makers in order to address the needs of those living with rare diseases. In 2019, the theme is 'Bridging health and social care'. This special day will focus on bridging the gap between medical, social and support services in order to tackle the challenges encountered by people living with rare diseases and their families.

For more information, please click here

Advances in Skeletal Muscle Biology in Health and Disease

6th - 8th March 2019

University of Florida, USA

This conference is well suited for informal interactions and the presentation of data that might be too preliminary for a larger audience. Overall, the goal is to facilitate advances in skeletal muscle biology through discussions that promote new ideas, research lines and collaborations.

Early Bird Discount Registration is currently available for the 2019 conference held at the University of Florida.

For more information, please click here

AFM - Telethon Scientific Congress in Myology

25th - 28th March 2019

Bordeaux, France

AFM-Téléthon is pleased to announce that their scientific congress in Myology will take place between 25th-28th March 2019. The congress will take place in Bordeaux, France.

More than ever, therapeutic innovation will be at the heart of this event in all its aspects: gene therapy, pharmacogenomics, gene surgery and others.

The successful research conducted in nerve and muscle physiology, and in neuromuscular disorders in general, as well as the multiplication of clinical trials involving hundreds of patients worldwide are now bearing fruit. The first effective treatments are now available for patients, and ongoing trials are more than ever synonym of hope and victories to come. We know it, a new era is opening up for our diseases that were regarded as incurable until recently, and MYOLOGY 2019 will reflect this change of paradigm for sure.

For more information, please click here

11th Annual Neuromuscular Translational Research Conference

4th- 5th April 2019

Newcastle, UK

The MRC Centre for Neuromuscular Diseases is pleased to announce the 11th Annual Neuromuscular Translational Research Conference will take place from 4th - 5th April 2019 at the International Centre for Life in Newcastle upon Tyne.

For more information on registration, the conference programme and abstract submission please click here

TACT Meeting

12th - 13th April

Glasgow, UK

Of the many promising research results presented at conferences, published in journals and hailed as the basis for possible future treatments and cures, few progress into clinical trial. Evaluating the therapeutic potential of drugs seemingly ready for this step is a challenge not only for the patients who build hope on preclinical results and for the potential funders and industry sponsors of the research, but also for the researchers themselves.

Established in 2009, the TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary international group of well recognized academic and industry drug development experts as well as representatives of patient foundations and institutional governmental scientific research centers, who meet twice a year to review and provide guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases with large unmet need, such as muscular dystrophies and amyotrophic lateral sclerosis (ALS).

The confidential and comprehensive review provides recommendations including go-no-go milestones, is independent of any funding stream however it may enable subsequent funding.

If you are interested in submitting an application to TACT, please contact Cathy Turner.

2019 MDA Clinical and Scientific Conference

13th - 17th April 2019

Orlando, Florida

The Muscular Dystrophy Association (MDA) is pleased to invite you to participate in the 2019 MDA Clinical & Scientific Conference on April 13-17, 2019, at the Hyatt Regency Orlando.

2019 will be an exciting year for MDA! We will converge our long-standing Clinical and Scientific conferences into our inaugural combined annual meeting, which will leverage MDA’s extensive reach into the scientific, clinical research, and clinical practice communities to bring together the world’s leading experts in neuromuscular disease.

As the most comprehensive neuromuscular disease meeting in the United States, it represents the full spectrum of scientific researchers, medical professionals, and decision makers. Our focus in 2019 will be to leverage the expertise of researchers, clinicians, and allied health professionals to accelerate drug development and advance best practices in care management across more than 40 neuromuscular diseases.

As an exhibitor and supporter, the 2019 Annual Meeting offers unequalled opportunities to be among other leaders in the healthcare industry to meet with and showcase your products and services to the neuromuscular disease scientific and medical community while you support MDA’s Mission – to transform the lives of individuals living with neuromuscular disease.

For more information, please click here