16th October 2015
Join us in Washington for the TREAT-NMD Conference 2015

Our first conference in the United States is fast approaching. With registrations still being taken we anticipate that this conference will be a sell out as our numbers are strictly limited.

Taking place in the historic Cosmos Club which is located in the DuPont Circle region of Washington DC the conference begins 6 December concluding on 8 December.

Entitled 'Growing the drug pipeline for neuromuscular diseases: optimising resources for clinical development of new therapies' the conference will bring together key stakeholders from the neuromuscular community.

Highlights of the conference include:

6 December

The first session chaired by John Porter will set the stage for the overall TREAT-NMD conference by providing recommendations for active participation of patients and caregivers in drug development. This will be followed by a session exploring the challenges of study design and animal model limitations.

7 December

The second day begins by focusing on the accelerating pace of clinical development for rare neuromuscular diseases and the unique sets of challenges and opportunities for all stakeholders. A variety of sessions chaired by clinicians, patient organisation and industry representatives will explore trial design, outcome measures, imaging and concluding with standards of care.

8 December

Patient registries have proven to be instrumental for clinical research in neuromuscular disorders. The morning session chaired by Hanns Lochmüller and Hugh Dawkins will set out the global picture. The conference will finish by exploring international trial readiness and present the importance of ensuring resources are efficiently utilized to bring the most effective drugs and to market to the most patients.

Register here

Executive Committee welcomes three new members
back to top    

We are delighted to announce the election of three new members to the TREAT-NMD Alliance Executive Committee.

Dr Nathalie Goemans has been duly elected to the Executive Committee filling the vacant academic position. Nathalie is a paediatrician and child neurologist and is Head of Clinic within the Department of Pediatric Neurology and Head of the Neuromuscular Reference Centre for Children at the University Hospitals Leuven in Belgium. She is also currently the Vice Chair of the TGDOC within TREAT-NMD.

Yuriko Oda has been elected to the Executive Committee filling one of two patient representative positions. She is the founder and President of the Japanese non-profit patient organization Patients Association for Distal Myopathy (PADM).

Petra Palmgren Lindwall will take up the second patient representative position. Since 2014 Petra has been the chairman of the Swedish non-profit patient organisation Swedish Research Foundation for Muscular Dystrophy (SMDF).

We would like to thank all TREAT-NMD members who voted in the election.

On behalf of the TREAT-NMD Alliance we want to thank all candidates for their interest in becoming a member of the Executive Committee, and we hope they will continue to support the activities of the TREAT-NMD Alliance in the future.

Last chance to submit abstracts for Myology 2016!
back to top    

The 5th International Congress of Myology will take place 14-18 March 2016. The best specialists from around the world will gather in Lyon, France to present and challenge their latest findings not only in fundamental research but also in clinical science and therapeutics.

Registration for the congress is now underway and early bird booking fees are in effect until 31 Jan 2016.

Please note the imminent deadline of Monday 19 October for abstract submissions.

Further details about the scientific programme along with important dates are available on the Myology 2016 website.

Recently updated DMD research overview now available in Spanish
back to top    

We are delighted to let you know that the TREAT-NMD research overview section for DMD which was recently updated to reflect the very latest information has now been translated into Spanish.

The TREAT-NMD research overview section for Duchenne muscular dystrophy (DMD) contains a succinct overview of therapeutic approaches that are in clinical development for DMD written in everyday language.

These pages have now been updated by Annemieke Aartsma-Rus to contain the latest clinical developments in the DMD field based on information presented at the Duchenne Parent Project Onlus meeting (Feb 2015, Rome, Italy) and the Duchenne Connect meeting (June 2015, Washington DC, US).

Financial support to Dr Aartsma-Rus by Duchenne Parent Project Onlus to attend the Duchenne Parent Project meeting in Rome and by the Duchenne Parent Project Netherlands to attend the Duchenne Connect meeting in Washington is gratefully acknowledged.

New myotonic dystrophy registry launched in Australia
back to top    

A new registry has been launched by the Office of Population Health Genomics for myotonic dystrophy patients in Australia.  The Australian National Myotonic Dystrophy (DM1) Registry has been developed to collect clinical and genetic information along with a novel patient reported questionnaire.

The Registry provides an important tool for clinicians and clinical trial sponsors to quickly identify patients suitable for clinical studies, particularly those therapeutic strategies that target specific genetic defects. In addition the registry aims to improve the care of DM1 patients through coordination of diagnosis and therapy in Australia.

The registry framework, developed by the Centre for Comparative Genomics, will also be adopted by the New Zealand myotonic dystrophy registry in the near future.

More information about the Australian National Myotonic Dystrophy (DM1) Registry is available on their website: nmdregistry.com.au

For any questions contact Leanne Lamont at the Office of Population Health Genomics.

International GNE myopathy registry - latest newsletter available
back to top    

The International GNEM-DMP registry has released its third newsletter and it is available to download from the registry website.

The newsletter is used as a way of informing patients and families, as well as doctors, about what is going on with the registry as well as containing updates from the study partner on anything else related to GNE myopathy.

This third issue contains the following articles:

- Genetic Testing and GNE myopathy
- Social Media and Clinical Trials – Tips For Getting it Right
- Food Rich in Sialic Acid – Is There any Benefit?
- Aceneuramic Acid (Sialic Acid) Extended Release (ER) Phase 3 Clinical Study Update
- Update  on Registry Component of the GNEM-DMP
- Los Angeles Patients Day - August 2015
- Get Involved: Clinical Study and GNEM-DMP Recruitment
- "My Journey so Far"- Marcia Bonome

See all of our newsletters on the International GNE myopathy registry

For more information on the GNEM-DMP Registry, including how to participate, contact HIBM@treat-nmd.eu

European Reference Networks - an update
back to top    

The Second Conference on European Reference Networks (ERNs) took place in Lisbon, Portugal on the 8-9 October 2015. The aim of the conference was to inform delegates about the processes surrounding establishing networks and raising awareness in general with healthcare providers. Delegates also discussed possible IT requirements for the new networks, eHealth solutions, and what can be learned from previously established networks and the added value ERNs could bring to EU Member States.

The conference was organized by DG Santé and hosted by the Portuguese Ministry of Health and was an event of the Luxembourg EU Presidency.

Coalition to Cure Calpain 3 requires new Scientific Director
back to top    

This part time post of Scientific Director will serve as the face of the Coalition to Cure Calpain 3 (C3) research mission. Working with the Board of Directors and the C3 Scientific Advisory Board, the Scientific Director will be responsible for developing strategies to craft, implement, and manage the research goals. Responsibilities include serving as the face of C3’s science advocacy, facilitating communication among various sectors of the research community (academic researchers, clinicians, biotechs, pharma, NIH, FDA, peer non-profit organizations), and communicating with families and donors. A key role of the Scientific Director will be to develop strategic relationships and partnerships with these other entities in pursuit of C3’s research goals.

Download further details to find out more about this part-time post.

EURORDIS requires Research Infrastructure Projects Manager
back to top    

Based in Paris as part of EURORDIS the main scope of the post includes the management of the RD-Connect project. Additional responsibilities include representing EURORDIS in several pre-clinical research consortia such as the European Platform for Rare Disease Registries. The successful candidate will also be responsible for representing EURORDIS in similar pre-clinical research networks which may be progressively added. The ideal candidate will have 3-5 years of relevant professional experience in a European or international academic environment, preferably in the non-profit, humanitarian or health sectors.

John Walton Muscular Dystrophy Research Centre seeks
Clinical Research Associate
back to top    

A cutting-edge clinical and translational researcher funded by the NIHR's Rare Disease programme is required. This full time post is available for up to 18 months and will be based at the John Walton Muscular Dystrophy Research Centre at Newcastle University led by Prof K Bushby, Prof H Lochmüller and Prof V Straub. The successful applicant will be able to participate in a range of different neuromuscular clinics across the MRC centre and gain exposure to state of the art diagnostic capabilities for neuromuscular diseases.

A medical degree and experience in medical history taking and patient assessment is required. The post will provide an opportunity to gain experience in clinical trials, paediatrics, neurology and clinical research along with laboratory work.

The post is tenable for 18 Months.

Sagol School of Neuroscience seeks postdocs
back to top    

Tel Aviv University’s Sagol School of Neuroscience will support prestigious post-doc positions for interdisciplinary research in neuroscience. They seek highly motivated candidates with a strong foundation in neuroscience that allows for independent interdisciplinary research, as well as an excellent publication record. Candidates should not be more than 5 years after receiving their doctoral degree. Priority will be given to non-Israeli candidates from universities outside Israel.

For further details please visit the job opportunities section of the TREAT-NMD website.

Submit an article
Past newsletters
16th October 2015
TREAT-NMD newsletter - 16th October 2015