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29th July 2016
World Duchenne Awareness Day 2016

Duchenne muscular dystrophy (DMD) is a genetically inherited condition, which causes muscle weakness in childhood and affects 1 in every 3,500 boys.

On September 7, 2016 the third World Duchenne Awareness Day will take place and will see stakeholders in the DMD community from around the world mark this special day with various events, all intending to raise the profile and awareness of DMD throughout the world. This year the campaign will be focused on Early Diagnosis programs.

To find out more about the day click here.

Nominations for Executive Committee
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As we announced in the last newsletter, there is an academic vacancy on the TREAT-NMD Executive Committee. Executive Committee members provide guidance to the secretariat and drive forward the TREAT-NMD workplan. Meetings of the Executive Committee are held monthly via a teleconference, with an annual face-to-face meeting.

Nominations can be on behalf of a colleague or self-nominations, and these should be sent to Stephen Lynn before the 31st August 2016.

Voting for this vacancy will take place in early September and the successful candidate will be announced in the September newsletter.

Face to Face Executive Committee Meeting
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TREAT-NMD will be holding a face-to face meeting of the Executive Committee on Thursday 6th October at 6.30pm. The meeting will take place in Granada, and is open to all TREAT-NMD members. If you are interested in attending the meeting please email us to let us know and full details and meeting agenda will be provided.

EUCOPE Declaration on Good Off-Label Use Practices (GOLUP)
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The prescription of medicines off-label, supported by strong medical and scientific evidence, is a recognised treatment solution where no other authorised treatment exists, or is available for a specific patient group or geography. Recent concerning events in Member States following the passing of legislation to promote the off-label use of medicines for economic purposes, highlight the importance of preserving the European regulatory framework to ensure the safety of patients. Off-label Use of medicinal products should only occur if the following criteria are met:

Presence of a severe, life-impairing or life-threatening condition;

Absence of authorised treatment or repeated treatment failure;

Absence of alternative treatments authorised for the condition;

The off-label use is supported by strong evidence in scientific literature;

The patient has been educated and has given his or her informed consent;

Presence of established reporting routes for adverse events and linked to off-label use.

These developments bring into question the robustness of the market authorisation framework, and may undermine the pharmacovigilance process and raise a number of liability issues, including for physicians and nurses. To raise awareness about these dangerous developments and promote a harmonised approach on this important issue in Europe, EUCOPE (EUCOPE – the European Confederation of Pharmaceutical Entrepreneurs) has decided to support the initiative of Professor Marc Dooms to identify and promote a number of clear criteria for good off-label use. These criteria stem from decades of research and clinical practice and serve to provide a clear framework on when and how the off-label use of medicinal products could safely take place. They do not purport to limit the off-label use, but intend to provide a harmonised approach for its occurrence, in order to maintain the highest levels of patient safety and minimise any adverse events. The aim of this initiative is to create broad consensus on the issue among main European stakeholders, notably physicians, patients, pharmacists, industry and research organisations.

ENMC Workshop: Myotonic Dystrophy - Developing
a European Consortium for Care and Therapy
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In July 2016 the 222nd ENMC workshop in Naarden, Netherlands brought together 24 experts (clinicians, scientists, regulators, industry and patient representatives) to discuss developing a European consortium for care and research in myotonic dystrophy. The organisers Baziel van Engelen, Hanns Lochmuller, Benedikt Schoser and Guillaume Bassez led the successful EU project OPTIMISTIC and wanted to build on the strengths of this collaboration and draw upon knowledge from the rest of Europe and colleagues in the US and Canada.

Clinical research in myotonic dystrophy is at a crucial point with two therapeutic approaches now in phase II trials. The workshop addressed trial readiness with discussions focussing on the need for clinically meaningful endpoints, harmonised registries and good quality natural history data. The consensus from the workshop was to reduce barriers to drug discovery and development in this condition through the establishment of a European Consortium that would share expertise and information to inform clinical trial design and patient treatment paradigms.

The full lay report is now available on the ENMC website

International GNE Myopathy Registry Newsletter
Multiple Languages Now Available
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The International GNE Myopathy Disease Monitoring Program (GNEM-DMP) registry has released its fourth newsletter in a range of different languages, that are available to download here.

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

This fourth issue of the newsletter contains the following articles:

• GNE myopathy Research and Clinical Trials with ManNAc at the US National Institutes of Health

• Patient Organisations and What They Do

• The Importance of Patient Advocacy for Rare Diseases

• Aceneuramic acid Phase 3 Clinical Trial Update (Ultragenyx) - (Update since the original release of this newsletter: This study is ongoing, but not recruiting participants).

• GNEM Registry Update

• Mobility and Use of Wheelchairs/Scooters in Registry Participants

• Participant Story: ‘My Journey So Far’ – Mark

Click here for more information on the GNEM-DMP Registry, including how to participate or email us.

Duchenne Muscular Dystrophy - Research Overview Updated
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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 Parent Project Muscular Dystrophy’s Annual Connect Conference (June 2016, Orlando, Florida, USA).

Financial support to Professor Aartsma-Rus by the Duchenne Parent Project Netherlands to attend the Duchenne Connect meeting in Orlando is gratefully acknowledged.

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Society of Neuromuscular Sciences Incorporated 42nd Annual Scientific Meeting and AGM. 8 - 12 August. Thredbo, Australia.

The Society of Neuromuscular Sciences Inc will include a variety of medical topics which will appeal to Specialists, Registrars, RMO's and General Practitioners.

45th European Muscle Conference. 2 - 6 September. Montpellier, France

The European Muscle Conference will cover all aspects of muscle research from basic to applied sciences. It offers an ideal forum for scientific exchange among specialists from different scientific disciplines and among young and senior scientists.

2016 Myotonic Dystrophy (MDF) Annual Conference. 15 - 17 September. Washington, DC. USA

The MDF Annual Conference is a community-focused event that brings together families, researchers and other stakeholders from all over the world. The program will build on the success of last year's event and will include:

  • The very popular Research Track featuring panels and presentations from industry and academic investigators on early-stage therapy development efforts
  • The Hill Day program, where you can meet with and influence congressional leaders about research and care
  • An expanded slate of physician and clinician-led sessions focused on disease understanding and management
  • New programs for networking with researchers, industry and other community members
  • A DM2 track
  • The Saturday night closing dinner party with music, dancing, food, and fun
  • The Resource Fair and much more
TREAT-NMD Summer Break
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This is our final newsletter before we take our traditional summer break and we would like to take this opportunity to thank everyone who has been involved with the newsletter throughout the last year.

We will return to your inboxes once again later on in September with news and information from the neuromuscular field. If you have any news or information that you would like to be considered for inclusion in forthcoming newsletters, you can always submit your article here. Don't forget you can always find out what we're up to by following us on Twitter.

We hope that those of you with vacations coming up have a great break and we look forward to contacting you all again in September.

Submit an article
Past newsletters
29th July 2016
TREAT-NMD newsletter - 29th July 2016
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