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11th February 2011
 
TREAT-NMD International Conference 2011: save the date and visit the new conference website!

The TREAT-NMD International Conference 2011 is due to take place from 8-11 November in Geneva, Switzerland. Co-organized with the Swiss patient organization Progena and bringing together industry, academia and patient groups as equal partners, this is a unique event in the neuromuscular calendar: a global conference focusing on the challenges facing translational research and therapy development and delivery in neuromuscular diseases today. Sessions ranging from natural history, care standards and outcome measures to gene therapy, antisense approaches, stem cells and emerging technologies will provide not only an update on the state of the art but also a forum for discussion of the challenges and steps to be taken to bring therapies to patients.

A dedicated conference website has just been launched and contains an overview of the program and venue as well as information about registration (which will be launched in March) and a call for poster abstracts. Please visit the new website for further details and mark the date in your diaries now!
 

 
 
 
New lay overview of SMA therapeutic approaches published
on TREAT-NMD website
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The DMD research overview written by Annemieke Aartsma-Rus of Leiden University Medical Centre received much positive feedback from patients and families after its publication on the TREAT-NMD website in 2008. Now Dr Eduardo Tizzano from Barcelona has created a similar overview to inform patients with spinal muscular atrophy (SMA) and their families about the approaches currently being investigated for the treatment of SMA. The comprehensive overview covers, in lay terms, the genetic aspects of SMA, the challenges faced by researchers working on treatments and cures, and the current therapeutic approaches that are being investigated. Like the DMD research overview, the production of the SMA text was facilitated by Maryze Schoneveld van der Linde of ENMC.

 
 
 
 
3rd TACT Meeting: Update
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The TREAT-NMD Advisory Committee for Therapeutics (TACT) held its third review meeting on the 15th - 16th January 2011, which brought together 21 multidisciplinary members of the committee. During the meeting the TACT members discussed two proposals submitted by industry researchers from the USA. Within 6 weeks of the meeting the committee will generate an assessment report providing recommendations to the researchers, and a non-confidential summary will be published on the TREAT-NMD website by 15 March.

The two proposals reviewed on this occasion were:

  1. Tetracycline Derivatives as SMN2 Splicing Modifiers for the Treatment of SMA – Paul Higgins, PhD, Director Inflammation Drug Discovery, Paratek Pharma, USA
     
  2. Therapy for Muscular Dystrophy by Inhibition of Mechanosensitive Ion Channels – Frederick Sachs, PhD, President, Rose Pharmaceuticals, USA
 
 
 
 
ENMC Workshop application deadline approaches
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A reminder that the deadline for applications to be submitted to the ENMC is 1st March for workshops to be held in the second half of 2011.

Applications should be via the forms available on the ENMC website.

Contact the ENMC office if you have any questions at enmc@enmc.org

Deadline: 1st March 2011

 
 
 
 
Upcoming meetings
 
Past newsletters
 
TREAT-NMD conference 2011
 
11th February 2011
TREAT-NMD newsletter no. 94
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