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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:


  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

Dr Higgins attended the meeting in Charlotte, North Carolina, USA in person to discuss his proposal. Dr Sachs was represented by the CFO of Rose pharmaceuticals, Jeff Harvey and by Dr Suchyna who is a research advisor on the application. This provided helpful additional background information for the committee as well as clarification of specific areas where the researchers were seeking specific advice.

Within 6 weeks of the meeting the committee will generate an assessment report providing recommendations. TACT hopes that this advice will be useful in effectively planning the next stages of the projects and strengthen the proposals if they are eventually submitted to funding organisations.

To help inform the community, TACT will also generate a non-confidential summary with input from the applicants, which will be made available via the TREAT-NMD website by 15th March 2011. However, parties interested in detailed information on TACT’s recommendations should request the full TACT report from the researcher.

Further information about the TACT process can be found throughout this section of our website.

The deadline for submissions for the next TACT meeting (15th-16th October 2011) is the 15th July 2011 and researchers should contact the TACT secretariat (emma.heslop@ncl.ac.uk and catherine.turner@ncl.ac.uk) to discuss their application before completing the full submission form.

The TACT Review Participants at the meeting in Charlotte were:

TACT core group:
Cristina Csimma, PharmD, MHP (Chair); Virdante Pharmaceuticals, USA
Didier Caizergues, PharmD DESS; GENETHON, France
Petra Kaufmann, MD MSc; NIH, USA (written report only)
Rudolf Korinthenberg, MD; University of Freiburg, Germany
John McCall, PhD; PharmMac LLC, USA
Elizabeth McNeil, MD; NIH, USA
Jerry Mendell, MD; Nationwide Children's Hospital, Ohio, USA
Kanneboyina Nagaraju, PhD MVSc; Children's National MC Washington, USA
Dominic Wells, PhD MA VetMB; Royal Veterinary College, London, UK

Additional TACT experts:
Arthur Burghes, PhD; Ohio state University, USA
Avital Cnaan, PhD MS; Children’s National MC, Washinggton, USA
Richard Finkel, MD; CHOP, USA
Pat Furlong, PPMD, USA
Miranda Grounds, PhD; The University of Western Australia, Australia
Sharon Hesterlee, PhD; PPMD, USA
Jane Larkindale, PhD; MDA, USA
Marie- Christine Ouillade; AFM, France
Kathryn Wagner, MD PhD; John Hopkins, USA
Meg Winberg, PhD; Independent consultant, USA

TACT secretariat:
Kate Bushby, MD MRCP FRCP MSc; Newcastle University, UK
Cathy Turner; Newcastle University, UK
Volker Straub, MD PhD; Newcastle University,UK

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 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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