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19th October 2011
Poster and speaker abstracts now available
on TREAT-NMD conference website

With less than three weeks before the conference begins, final preparations for the TREAT-NMD conference are now almost complete. We have been delighted with the quality of the poster abstracts that we have received and all abstracts are now available to view on the conference website.

The final programme is now also complemented by a complete list of speaker abstracts that demonstrate not only the strength of the programme but the high calibre of speakers at the conference. Click each speaker's name to view the abstract of their talk.

Final places are still available for those wishing to attend, and we would recommend interested parties to visit the registration section.

The challenges of systemic delivery of AAV for neuromuscular
gene therapy: workshop report
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As part of their preclinical work within TREAT-NMD, TREAT-NMD partner Généthon arranged a one-day meeting of experts to discuss issues surrounding the preclinical evaluation of adeno-associated virus (AAV) vectors as a mechanism for systemic delivery of neuromuscular disease therapies. This is a critical step when moving from the research and development phase towards clinical studies. The many challenging questions surrounding mode of administration, animal models, biosafety issues and immune responses have meant there is still a lack of consensus between experts, and the primary goal of the meeting was an attempt to establish common ground. The meeting was split into four round-table sessions:
1: Non-regulatory preclinical studies: route and dose rationales, safety pharmacology studies. What should be covered before toxicology studies?
2: Regulatory toxicology and biodistribution of the product: How to choose the best model(s) and protocol(s) in animals to forecast worst-case tox effects?
3: Surgical (isolated limb local perfusion) potential side-effects and local toxicity: What is the best animal setting for a pertinent surgical model?
4: Immune response / immunotoxicology. What do we know about the potential immune-related side effects on efficacy and safety? Immune suppression?

A meeting report and conclusions are available by clicking the "more" link below.

CARE-NMD European DMD patient survey underway
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The CARE-NMD survey of patients and families living with Duchenne muscular dystrophy (DMD) is now underway in all seven of the project's partner countries.

If you are living with DMD in Bulgaria, the Czech Republic, Denmark, Germany, Hungary, Poland or the United Kingdom, and are registered in your National Patient Registry, you should have received an invitation to take part in the survey. If you are registered but have not yet received an invitation, please get in touch with your National Patient Registry directly.

CARE-NMD aims to improve the quality of care for DMD, and is surveying both those living with the condition and healthcare professionals with a view to identifying and tackling barriers faced by healthcare professionals in adopting the DMD Standards of Care.

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EVELAM, the Escuela de Verano Latino-Americana de Miologia or Latin American Summer School in Myology is an annual short course in myology modelled on the Paris summer school that rotates through the countries of South America. Led by Andoni Urtizberea (Paris Summer School), Norma Romero (Institut de Myologie), Alberto Rosa (University of Cordoba) and Jorge Bevilacqua (University of Chile), each course is arranged with the active support of local organizers in its host country and draws in 100-120 participants on average.

This year's course will take place in Sao Paulo, Brazil, from 8-10 December 2011 and the local organiser is Dr Edmar Zanoteli.

Upcoming meetings
Past newsletters
TREAT-NMD conference 2011
19th October 2011
TREAT-NMD newsletter - 19th October 2011
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