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29th July 2011
TREAT-NMD conference: abstract submission deadline extended!

We have been delighted by the number and variety of abstract submissions we have received for poster presentations at our conference in Geneva from 8-11 November 2011. In response to a number of requests to allow those with vacation commitments and those who haven't had a chance to register for the conference to submit an abstract, we have extended the deadline for abstract submission to Thursday 1st September.

The conference will be an exciting opportunity for anyone engaged in translational research in neuromuscular diseases to present their work whilst earning 18 CME credits at the same time.

Posters may cover any relevant area from novel basic research to new technologies in neuromuscular disease research, translational research and clinical trials, natural history studies, outcome measures research and development, or clinical aspects of patient care and management. In addition, posters focusing on the patient perspective and the social and ethical aspects of living with a neuromuscular disease and taking part in clinical research are welcomed.

All abstracts will be reviewed by the programme committee after the closing date, and applicants will be informed of the outcome of the review by the end of September 2011. Accepted abstracts will be published in the conference book provided to all delegates and will also appear on the conference website.

Please note: the deadline for late-breaking news submissions is Friday 30th September.

TACT - Current round of applications announced
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TACT, the TREAT-NMD Advisory Committee for Therapeutics, is pleased to announce that based on the preliminary review of the applications and inquiries received as of July 15th, the closing date for the fourth TACT review meeting, the following three applications will be reviewed at the TACT meeting scheduled for 15th-16th October 2011 in Lisbon, Portugal:

1) Marc B. Blaustein, MPP, Halo Therapeutics, LLC. Newton, Massachusetts, USA.
A randomised, double-blind, placebo-controlled, multiple-dose, dose-escalation study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of HT-100 in patients with Duchenne muscular dystrophy.

2) Fabrizio Dolfi, MD, PhD, NicOx SA. Sophia Antipolis, France.
A 6-month multicenter, randomised, double-blind, placebo-controlled, Phase IIa proof of principle study of naproxcinod (HCT 3012) 750 mg bid in patients with Becker Muscular Dystrophy.

3) Chris N Airriess, PhD, California Stem Cell. Irvine, California, USA.
Human Embryonic Stem Cell Derived Motor Neuron Progenitors for the Treatment of Motor Neuron Disease.

As with previous reviews, TACT will generate a detailed report to the applicant within 6 weeks following the meeting. A general 'non-confidential' report summary, developed in collaboration with the applicant, will be available via the TREAT-NMD website within 8 weeks following the meeting at www.treat-nmd.eu/TACT/.

Dates for the subsequent TACT meeting have been confirmed as 28th-29th April 2012 and individuals interested in potentially submitting an application for this meeting should send an expression of interest to the TACT secretariat - Emma.Heslop@ncl.ac.uk - as soon as possible.

New resource on cord blood banking
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Following on from the popular TREAT-NMD resource on stem cell tourism, we have expanded the guide to include private cord blood banking. Private blood banks work on the hope that there will be a therapy in the future for muscular dystrophy using stem cells and that storing cord blood from an unaffected sister or brother to use in such treatment will be useful. Families need to balance this possibility against the financial cost of using cord blood banks.

TREAT-NMD supports the development of publicly organised cord blood banks, where cord blood is donated without payment for the benefit of anyone who needs it. The TREAT-NMD guide can be found via the "more" link below.

Translations update: Family Guide for DMD
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Since its launch last spring, the Family Guide for the Diagnosis and Management of Duchenne Muscular Dystrophy has been very well received, and we have been delighted with the response to the call for translations.

Thanks to the hard work of volunteers worldwide, the guide is now available on the TREAT-NMD website in 18 languages, including: Bulgarian, Chinese (simplified and traditional), Czech, Danish, English (UK and US versions), Finnish, French, German, Hebrew, Hungarian, Lithuanian, Polish, Romanian, Russian, Serbian, Slovenian, Spanish and Turkish.

In addition, translations of the guide into other languages (such as Arabic, Greek, Japanese, and Swedish) are well underway.

TREAT-NMD would like to thank all the volunteers so far for their efforts, as making translations available in as many languages ensures it is accessible to all DMD patients and families.

We would also like to put a call out for volunteers to translate the family guide into languages not yet covered - a full list of completed and in-progress translations is available on the website.

The family guide to SMA care is also currently available in 13 languages, and we would be delighted to hear from anyone who would be able to translate this into missing languages as well. Later this year, the family guide based on the academic article on CMD care standards will also be published.

If you are able to help, or can tell us about other translations so that we can keep a comprehensive list of translations up to date, then please contact Sunil Rodger at the TREAT-NMD Co-ordination Office.


Newsletter 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.

September will see the TREAT-NMD newsletter return to your inbox on a different day of the week. To enable you to keep up to date with our news while you are in the full flow of the working week we have decided to send out our newsletter every second Wednesday beginning on 7th September.

If you have any news or information that you would like to be considered for inclusion in forthcoming newsletters, please visit our site updates section to submit an article for consideration.

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.

Upcoming meetings
Past newsletters
TREAT-NMD conference 2011
29th July 2011
TREAT-NMD newsletter - 29th July 2011
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