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5th October 2011
Industry Forum at the TREAT-NMD International Conference, Geneva

In the run-up to our conference in Geneva we again highlight one of the conference sessions. The industry forum will be led by Joseph Irwin, a regulatory consultant who also had a son with SMA Type I, and Dr Ed Connor, Director, Office of Investigational Therapeutics, at the Children’s National Medical Center and previously Executive Vice President and Chief Medical Officer at MedImmune, Inc. The industry forum will include representatives from various companies who will be in Geneva for the conference to discuss a number of concerns and issues facing the pharmaceutical industry. The issues to be covered include;

  • Patient recruitment
  • Validity and utility of n=1 studies
  • Access to study drug outside a clinical trial
  • Regulatory hurdles
  • Restrictions set by ethics committees and IRBs

During the forum industry colleagues involved in previous or ongoing studies will be asked to address these issues and how they affect their ability to run successful clinical trials. The forum will close with an overview of what we have learnt and what still needs to be addressed to support efficient and effective clinical development programmes.

The TREAT-NMD International Conference programme has now been finalised and is available on the conference web site, along with our second announcement that includes further information about the conference and how to register. We look forward to meeting you in Geneva for what will be an exciting and stimulating round-up on the state of translational research for neuromuscular diseases.

We also acknowledge the Starling Hotel and Conference Centre as our official hotel partner in Geneva.

UK National Institute for Health and Clinical Excellence
officially endorses development process of
standards of care for DMD
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The National Institute for Health and Clinical Excellence (NICE) in the UK has officially endorsed the development process underpinning the DMD Standards of Care, which set out international consensus guidelines for the care for patients with Duchenne muscular dystrophy.

The recognition of the Care Standards as "Accredited Evidence" by NICE marks the culmination of a process led by the Muscular Dystrophy Campaign and Professor Kate Bushby, which certifies the clarity and rigour of development of the guidelines. It also recognises that the information is accessible and available to a broad audience, through the production of a Guide for Families which has now been translated into 20 languages.

Professor Kate Bushby said, "We are delighted that the Duchenne Standards of Care have been recognised by NICE as Accredited Evidence. Both the Muscular Dystrophy Campaign - with whom I submitted the application - and I are committed to ensure that all patients with Duchenne muscular dystrophy have access to the high standards of care set out in the document to extend their life expectancy and protect their quality of life. This is a vital step to enable commissioners to provide appropriate services for people affected by muscle disease."

This recognition, the first for a neuromuscular condition, is a very important step in improving access to high quality care for DMD. It enables health care providers to have confidence that the guidelines were developed impartially and to a high standard. The Muscular Dystrophy Campaign is now looking into the possibility of gaining accredited evidence status for the Consensus statement for the care of spinal muscular atrophy.

A full statement by the Muscular Dystrophy Campaign is available on their website.

EuroBioBank Network Annual Meeting Report for 2011
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The 9th EuroBioBank (EBB) Network Annual Meeting, organised by the European Organisation for Rare Diseases (EURORDIS) in Paris on 8-9 September 2011, was attended by 20 participants representing most member biobanks.

EuroBioBank, the European network of DNA, Cell and Tissue Banks for Research on Rare Diseases, was established in 2001 and has been coordinated by EURORDIS since its inception, with continued EU funding through the TREAT-NMD grant since 2007. It is a unique network currently composed of 16 biobanks from 8 EU countries, with a focus on neuromuscular research. Its objective is to increase availability, facilitate access to and use of quality human biomaterials for research on rare and neuromuscular diseases.

The meeting participants discussed the future of the network, new grant applications, quality certification for biobanks, fee structures for commercial requests, and collaboration with US initiatives.

The full meeting report can be found on the TREAT-NMD website by clicking the 'more' button.

CARE-NMD launches European DMD patient survey
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The CARE-NMD survey of patients and families living with Duchenne muscular dystrophy (DMD) is now live for those living in six European countries - Bulgaria, the Czech Republic, Denmark, Germany, Hungary, and the United Kingdom - with the survey due to launch in Poland soon.

The project, which aims to improve the quality of care for DMD, is conducting the survey to ascertain the availability of high-quality DMD care and the quality of life experienced by both patients and their families. It asks about care in line with the international consensus care standards for DMD, as well as using the validated health survey tools PedsQL, KIDSCREEN, WHOQOL-BREF and SF-36.

DMD patients and their families in all countries covered by the project are strongly encouraged to take part in the survey: if they are registered on their respective National Patient Registry, they should have received an invitation. As the details of how to take part are slightly different in each country, more information can be found in each country section of the CARE-NMD website.

Over the coming months a survey of clinicians in all seven countries will also be conducted by the project, to gain a better understanding of any barriers faced by healthcare professionals in adopting the DMD Standards of Care.

Correction 05/10/11: a factual error relating to the health survey tools has been corrected

1st Belarusian Neuromuscular Symposium
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The 1st Belarusian Neuromuscular Symposium will take place in Minsk, Belarus, on 13 October. It consists of a day of lectures from international and Belarusian  specialists (with simultaneous interpretation between Russian and English), including broad-reaching overviews of neuromuscular disease in children and adults and presentations on diagnostics, outcome measures, patient registries and networking. The day concludes with an interactive case presentation session in which doctors present challenging cases and open discussion takes place to allow the experts to share their knowledge and experience.

Upcoming meetings
Past newsletters
TREAT-NMD conference 2011
5th October 2011
TREAT-NMD newsletter - 5th October 2011
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