unsubscribe | view this newsletter online | full newsletter
27th October 2017
Last 30 tickets! TREAT-NMD Conference

The 5th International TREAT-NMD conference, taking place from 27-29th November 2017 at the Konzerthaus, Freiburg, is shaping up to be a very informative event and will be attended by international key opinion leaders and stakeholders of TREAT-NMD, including investigators, patient organizations, and clinicians. Our meeting will address progress and lessons learned in the area of translational medicine in inherited neuromuscular diseases and plan for the delivery of future therapies to patients.

The full conference programme can be viewed here.

Due to increasing demand, there are less than 30 places available for delegates, so it is imperative to book now to avoid disappointment. To register, please click here or for further information, please contact Anne Oyewole (anne.oyewole@newcastle.ac.uk)

International GNE Myopathy Registry Newsletter (9th Edition)
- Multiple Language Versions Available
back to top    

The International GNE Myopathy Disease Monitoring Programme (GNEM-DMP) registry has released its ninth newsletter in a range of languages. These are now available to view online or download at the  GNEM-DMP or TREAT-NMD website.

The newsletter is used as a way of providing information and updates to patients, families and doctors about the registry and other activities related to GNE Myopathy.

Available Languages: Arabic, Bulgarian, Chinese, Dutch, English, Farsi, French, German, Hebrew, Italian, Korean, Portuguese, Spanish, Turkish.

This ninth issue contains the following articles:

  • Findings of the UX001-CL301 Clinical trial
  • Highlights if the 2017 Neuromuscular Disease Foundation Symposium on GNE myopathy
  • Italian GNEM patient meeting hosted by National Associazione Gli Equilibristi
  • GNE Myopathy through the eyes of caregivers.


For more information on the GNEM-DMP Registry please visit www.gnem-dmp.com or email GNEM@treat-nmd.eu

SMA Family Guide - Bahasa Malaysia Translation
back to top    

Since its launch, the Family Guide for the Diagnosis and Management of Spinal Muscular Atrophy (SMA) has been very well received by the community, and we are delighted to announce the availability of the Bahasa Malaysia version which is now available to download from our website.

The family guide to SMA care is currently available in 20 language - to view these please click here.

TREAT-NMD would like to thank all volunteers who have contributed to making translations available in a number of languages. This has helped to ensure information about standards of care are accessible to SMA patients and families.

Genome Editing Technology CRISPR/Cas9 Remove Toxic RNA
that Causes Myotonic Dystrophy
back to top    

A new version of the genome editing technology CRISPR/Cas9 has been used to remove the toxic RNA that causes myotonic dystrophy. This study which was conducted by scientists in the department of Cellular and Molecular Medicine at the University of California is an important proof-of-principle, that shows that the technique works in cells in the laboratory. Overall, the findings indicate the therapeutic potential for the CRISPR/Cas9 system to broadly reverse the molecular pathology of myotonic dystrophy type 1 and 2, however further research is still needed before it can be tested in humans.

To view the full publication please click here

2018 Expert Patient and Researchers Eurordis Summer School
back to top    

Opportunity to participate in the 2018 Expert Patient and Researchers Eurordis Summer School

In 2018 there will be another Expert Patient and Researchers (ExPRESS) Eurordis Summer School (June 11-15 2018, Barcelona, Spain). As before, this hands-on summer school is a unique opportunity open to patient representatives and academic researchers to learn about drug development, from clinical trials to the regulatory system, health technology, orphan drug legislation and ethical aspects. For more information, please click here. Multiple members of the TREAT-NMD executive committee and secretariat are alumni and highly recommend this course to anyone working in translational research.

If you are interested in participating, you can apply online (deadline November 30 2017).

Note that travel and hotel costs have to be borne by academic participants but that fellowships are available if needed.

Job Vacancy- Action Duchenne
back to top    

Salary dependent on qualifications and experience

The UK DMD Registry was developed by Action Duchenne in 2007 and acts as a database for all those living with Duchenne or Becker Muscular Dystrophy (D/BMD).  Patient registration is an online process that is voluntary and confidential. The Registry collects information including genetic diagnosis, some clinical function information and interventions.

We are looking for an analytical individual with a sound knowledge of genetics to support the DMD Registry Curator and to lead on the Registry’s future development and planning. To ensure the collection and analysis of data is accurate, efficient and to implement improvements to future-proof the Registry. It is an exciting time for Duchenne translational research as there are around 40 clinical trials taking place now, with new trials planned across the UK.

See full job description.

To apply, please send your CV with a brief covering letter.  Closing date is 24th November 2017 by 5.00pm.  Only suitable candidates will be contacted for a brief, informal telephone interview on the 27th November between 11.00am and 2.00pm.  Final interviews will take place on the 11th December.

Please send all applications to Angela Stringer, Action Duchenne, Epicentre, 41 West Street, London E11 4LJ or email

Next TREAT-NMD Newsletter - December
back to top    

Due to the forthcoming TREAT-NMD Conference (27-29th November), the next issue of the TREAT-NMD Newsletter will not be published until mid-December.

We would like to continue to encourage you to submit articles, publications, meetings etc. across all neuromuscular disorders using our online form.

If you have any questions please contact us at info@treat-nmd.eu

Upcoming Meetings and Events
back to top    

The Neurotechnix 5th International Congress

30-31 Oct 2017

Madeira, Portugal

The Neurotechnix 5th International Congress will be a meeting point of multidisciplinary teams, of both biomedical and engineering professionals, academics and practitioners. It will promote translational discussions on how technology can meet the needs of both clinical practitioners and persons with neurological disorders.


Action Duchenne 15th International Annual Conference

10-12 Nov 2017

Birmingham, UK

The Action Duchenne 15th International Annual Conference will be taking place on the 10th of November 2017 - 12th of November 2017 at Hilton Birmingham Metropole.​ The conference will ​once ​again ​bring ​Duchenne ​& ​Becker ​families ​together ​with ​experts ​in ​a ​range ​of ​fields ​to ​tackle ​Duchenne ​& ​Becker ​muscular ​dystrophy.
If you are interested in the event then please contact Angela Stringer at angela@actionduchenne.org or on +44(0) 20-8556-9955


5th Chinese International DMD Conference

11 - 12 Nov 2017

Beijing, China

The 5th Chinese International DMD Conference will bring together various stakeholders from the whole China neuromuscular community and beyond including neurologists, physiotherapists and psychiatrists. So far, over 30 local neurologists and 100 DMD families have already registered for this meeting, and Speakers from around the world will provide an international aspect for medicine development and clinical Trials.

If you are interested in the event then please contact Jan Wang at chinadmd.wz@gmail.com or Chinadmd.org@gmail.com


1st International Conference on Imaging in Neuromuscular Disease

19 - 21 Nov 2017

Berlin, Germany

Registration is still open for the 1st International Conference on Imaging in Neuromuscular Disease. The conference programme will feature internationally-recognized speakers and will highlight developments and advances in all aspects of muscle imaging with sessions on Diagnostic Muscle Imaging, New Imaging Techniques and Quantitative Muscle Imaging.

Due to increasing demand, there are less than 10 places available, so book now to avoid disappointment.


5th TREAT-NMD International Conference

27 - 29 Nov 2017

Freiburg, Germany

The 5th TREAT-NMD International Conference is a fantastic opportunity for patients, academics, clinicians, patient registry curators and industry representatives, indeed all TREAT-NMD stakeholders, to get together to network, learn and exchange ideas about the latest translational research.
To view the programme click here or to register please visit our dedicated conference website.

27th October 2017
TREAT-NMD Newsletter 27th October 2017
unsubscribe | view this newsletter online | full newsletter