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25th March 2011
TACT advice: summary reports from January meeting now available online

TACT evaluated two proposals at the review meeting on January 15-16th 2011. The TACT reports with recommendations were sent to the applicants within 6 weeks of the meeting and the corresponding non-confidential reports, approved by the applicants, have now been uploaded to the TACT webpage on the TREAT-NMD website.

TACT evaluated two proposals at the review meeting held on January 15-16th 2011 in Charlotte, US. These were:

  • ‘Tetracycline Derivatives as SMN2 Splicing Modifiers for the Treatment of SMA’  submitted by Dr Paul Higgins, Paratek Pharmaceuticals, USA
  • ‘Therapy for Muscular Dystrophy by Inhibition of Mechanosensitive Ion Channels’, submitted by Dr Frederick Sachs, Rose Pharmaceuticals, USA

The TACT reports with recommendations were sent to the applicants within 6 weeks following the meeting. The corresponding non-confidential reports, approved by the applicants, have now been uploaded to the TACT webpage on the TREAT-NMD website. Anyone interested in the full TACT report should contact the applicants directly:
Paul Higgins, PhD
Dr. Frederick Sachs

TACT gratefully acknowledges the financial support of the patient organization PPMD towards the costs of the January meeting. 

Role of TACT: the TREAT-NMD Advisory Committee for Therapeutics is an expert multidisciplinary body that provides the neuromuscular community (clinicians, researchers, patient advocacy groups and industry) with independent and objective guidance on advancing new therapies for neuromuscular diseases. Its goal is to position the potential therapy along a realistic pathway to eventual clinical trial and registration by evaluating preclinical data as well as drug development considerations that are crucial for the conduct of studies that generate meaningful data. Further details of the TACT process and the benefits of a TACT review can be found on the TREAT-NMD website

The current chair of TACT, Cristina Csimma, explained its value to therapeutics research: “The strength of TACT is its broad scientific and development expertise coupled with the rigour and independence of its review process, performed in a truly global context. This committee can provide objective and constructive guidance from world experts in the field that will both help researchers focus on areas for development and ensure the wider community is better informed about the readiness of new therapies for the next step.”

The next TACT review meeting will be held in Lisbon, Portugal on 15-16th October 2011 (immediately prior to the 2011 WMS meeting). Anyone wishing to submit a proposal for this meeting should contact the TACT secretariat via emma.heslop@ncl.ac.uk in order to discuss this further.

The deadline for applications is 15th July 2011.

Recent additions to the registry of outcome measures
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During the last few weeks a number of new records have been added to the Registry of Outcome Measures (ROM).  You can access a list of recent additions on www.researchrom.com/ra. Some highlights are provided below for your information.
Upper Limb Functional Items: For anyone interested in upper limb functional items we've added two new stage 1  records (Name + Reference) in this area: the Assisting Hand Assessment (AHA) and the Manual Ability Classification System (MACS).  Both records link to the OM websites which contain a range of information from the background of development, to publications concerning vailidity, and much more.
Functional Rating Scales: For those interested in this area, the Muscular Dystrophy Functional Rating Scale was added in February.  This is a stage 3 record (substantial descriptive summary) and includes a link to the key reference.
Devices: For those interested in measurements associated with walking, a new device has also been added.  Known as DataGait, the device measures gait, with generic application across many diseases.  This stage 3 record includes links to additional information as well as the distributors contact details.
Ongoing Work: For anyone interested in myotonic dystrophy, inclusion body myositis and fascioscapulohumeral dystrophy, ROM is presently supporting international collaborative efforts towards the identification of OMs and related information that we hope will serve to inform future discussions and decisions in the selection of OMs.  The work on IBM and FSHD is presently focussed mainly at functional items/scales,  MMT an QMT.  Work in DM has expanded slightly beyond this with the wealth of information being volunteered by the various experts contributing to the discussion.  Contributions to this discussion are being added and published via the relevant branch on the Review/Selection tree and we welcome feedback from all experts interested in these areas.  Please feel free to have a look at the information being gathered www.researchrom.com/review and let us know if you think there is something missing: Registry Coordinator Joanne.Auld@iop.kcl.ac.uk.
Our thanks! to those individuals and groups who have taken the time to contribute these and other records to ROM.  Your support and effort is very much appreciated.
New Contributions: We continue to seek the contributions of experts from around the world.  If you know of an OM that is used or potentially suitable for application in NMD and is not yet published in ROM please tell us about it.  All it takes to publish a stage 1 record is the official name of the OM and a key reference or official web site.  Just copy and paste the following information into an email with the subject title: New OM for ROM, fill in the missing details, and send it to the Registry Coordinator Joanne.Auld@iop.kcl.ac.uk:
OM Name:
OM Abbreviation (if known):
Your Name (including title: Dr, Prof, etc):
Your Institution:
We'll check the details, publish references in ROM, and when a record is published we'll add you and your organisation to our list of contributors.
If you've got the expertise in a particular OM and would be willing to contribute more information, we'd love to hear from you and to set you up with an official user account.  On average, those with a detailed knowledge of an OM can complete a stage 2 record in about 30 to 45 minutes.

Russian Spring School of Myology: Moscow, 18-21 April
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Venue: Russian Academy of Civil Service (Российская академия государственной службы), Moscow (www.rags.ru)

18-21 April 2011

Download the program here.

This is a specialist neuromuscular training course for healthcare professionals in the style of the myology summer schools that are held every year in Paris and London. Its aim is not only to provide updates on different aspects of neuromuscular disease care and research but also to encourage networking and communication between different muscle centres and disciplines, both within Russia and between Russia and other countries.

Presentations are given by expert clinicians from Russia and abroad, in Russian or English with simultaneous translation. Workshop and case study sessions enable extensive interaction between participants and allow the experts to share their knowledge and experience in resolving challenging real-life cases.

The Moscow 2011 event is co-organised by the patient organisation Nadezhda (the Russian MDA), the Moscow Region Paediatric Psychoneurological Hospital No.1 and the Moscow Research Institute of Pediatrics and Child Surgery, in collaboration with the ENMC for TREAT-NMD. Andoni Urtizberea is the international clinical lead.

Registration is open to clinicians from across Russia and other Russian-speaking countries. For further information and details of how to register, contact:

Svetlana Artemieva or Andoni Urtizberea

Submit your WMS poster abstracts by 31 March!
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Abstracts for the 2011 WMS congress in Portugal need to be submitted by 31st March at the latest to be considered for inclusion. Your abstract should contain a maximum of 300 words or 2000 characters.

Poster topics should fall into one of the following areas: Duchenne and Becker muscular dystrophy and associated dystrophinopathies; New developments in spinal muscular atrophies and hereditary neuropathies; Advances in therapy of neuromuscular disorders or Advances in all other neuromuscular fields.

Please visit the WMS 2011 website for further details on how to submit your abstracts.

Myology 2011 Congress:
Deadline for late-breaking news submission 15 April
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The Myology Congress 2011 will take place from May 9 to 13 in Lille, France, at the Lille Grand Palais. The scientific organization committee welcomes late breaking news, ideally with hitherto unpublished data having a significant impact in the field of myology, to be included in the "surprise box" session on the final day of the conference (Friday 13 May 2011).

To have your late-breaking news considered for this session, please send a short overview to Dr Andoni Urtizberea, andoni.urtizberea@hnd.aphp.fr

Upcoming meetings
Past newsletters
TREAT-NMD conference 2011
25th March 2011
TREAT-NMD newsletter no. 97
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