Downloads
All the downloadable files from the whole site are also available from this section.
- Mutation-specific approaches- Mutation-specific therapies
About TREAT-NMD- Research Approaches for a Therapy of Duchenne Muscular Dystrophy
- PPMD Annual Connect Conference_Philadelphia 12 - 14 July 2007
- Interview with Prosensa July 2008
- Interview with PTC July 2008
- Interview with Prosensa July 2008
- Interview with PTC July 2008
- Exon Skipping Report 2009
- Exon Skipping Report 2009 - Japanese
- Exon Skipping Report 2009 - Spanish
- Exon Skipping Interviews with Kate Bushby, Hans Schikan and Judith van Deutekom
- Exon Skipping Interviews with Kate Bushby, Hans Schikan and Judith van Deutekom - German
- Exon Skipping Report 2009 - German
Animal Models- Mammalian animal models for Duchenne muscular dystrophy
Become a member!- Membership application form
- Members Charter
Biobanks- EuroBioBank leaflet
- EuroBioBank Network charter
DMD- DMD care recommendations
- K Bushby et al. The multidisciplinary management of DMD
- Corticosteroids in DMD - ENMC workshop report
- DMD care recommendations - French
- DMD care recommendations - Dutch
- DMD care recommendations - Italian
- DMD care recommendations - German
- DMD care recommendations - Czech
DMD and SMA patient registries in Turkey- Registry Brochure in Turkish
DMD care standards- DMD - Interim Recommendations - Czech
- DMD - Interim Recommendations - Dutch
- DMD - Interim Recommendations - English
- DMD - Interim Recommendations - French
- DMD - Interim Recommendations - German
- DMD - Interim Recommendations - Italian
- DMD - Interim Recommendations - Serbian
DMD patient registry in the Czech Republic and Slovakia- Characterization of the DMD/BMD patient population in Czech Republic.
Duchenne Research Reports - Guenter Scheuerbrandt- PPMD Philadelphia 2007
- Prosensa interview 2008
- PTC interview 2008
- Research Approaches Report 2008
- Exon Skipping Report 2009
- Exon Skipping Report 2009 - Japanese
- Exon Skipping Report 2009 - Spanish
- Exon Skipping Interviews with Kate Bushby, Hans Schikan and Judith van Deutekom
- Exon Skipping Interviews with Kate Bushby, Hans Schikan and Judith van Deuttekom - German
- Exon Skipping Report 2009 - German
- Exon Skipping Report 2009 - October Update
EMEA DMD- Meeting Agenda
- Press Release 02.10.09.
- 149th ENMC WS report- Systemically delivered AON
- Multiexon skipping
- AON analogs for exon 46
- Harmonisation of OM for DMD
- Reliability of NSSA
- Theoretic Applicability of antisense-mediated exon skipping
- Therapeutic potential of AON exon skipping
- Skipping towards personalized molecular medicine
- Reliable surrogate Outcome Measures in multicentre clinical trials of DMD
- A Renaissance for AON drugs in Neurology
- B Wilcken Trial 2001
- Evidence-Based Medicine for Rare Diseases
- Edwards Trial 1997
- Small Clinical Trials
- List of Participants
EMEA-TREAT-NMD SMA outcome measures workshop- Speakers portfolio
- Clinical Presentation Ambulatory SMA - K. Zerres
- SMA Information Dossier
- Scientific Consensus on OM for non-ambulant SMA - E. Mercuri
- Scientific Consensus on OM for SMA Type I- R. Finkel
- Final Agenda 13.10.08
- TREAT-NMD/ EMEA Meeting Agenda
- Clinical presentation SMA Type I- J. Kirschner
- Future Perspectives for Clinical Trials in SMA- F. Muntoni
- What is SMA- E.Bertini
- Agency expectations on OM- R. Elbers
- Patient Perspective- P. Streng
- Scientific Consensus on OM SMA Type III- J. Florence
- Overview of Biomarkers in SMA- L.Simard
- Scientific Consensus on Biomarkers for SMA- FD.Tiziano
- Electrophysiology as a biological marker - K.Swoboda
- Clinical presentation of non-ambulant SMA- P.Kaufmann
- Introduction - V.Straub
- Introduction - C. Joyce
Experimental Protocols- Behavioral and locomotor measurements using open field animal activity monitoring system
- Whole body tension measurements
- The recovery score to evaluate therapy efficacy in NMD: a common, quantitative and comparative scoring system
- Use of treadmill and wheel exercise for impact on mdx mice phenotype
- Use of grip strenght meter to assess the limb strenght of mdx mice
- Measuring isometric force of isolated mouse skeletal muscles in situ
- Respiratory system evaluation
- Use of patch-clamp technique to study voltage-independent cationic channels in normal and dystrophic skeletal muscle fibres
- Non-invasive echocardiographic assessment of cardiac function in the mdx mouse model
- Measuring isometric force decrement after eccentric contraction in vivo in GRMD dogs
- Measuring tibiotarsal joint angle to assess contractures in vivo in GRMD dogs
- Measuring tetanic isometric force at the tibiotarsal joint in vivo in GRMD dogs
- Left ventricular pressure-volume (P/V) loops
- Use of treadmill and wheel exercise to assess dystrophic state
- Measuring isometric force of isolated mouse muscles in vitro
- Workshop report in Neuromuscular Disorders - preclinical testing for DMD
- Quantitative determination of muscle fiber diameter (minimal Feret’s diameter) and percentage of centralized nuclei
- Determination of hydroxyproline content as a measure of fibrosis in nondystrophic and dystrophic skeletal muscle
Information for industry and researchers- Registries Charter
International Conference- Conference Flyer and Programme
Introduction- Conference Flyer
- Guenter Scheuerbrandt - Conference Report
Meeting reports archive- Research Approaches for a Therapy of Duchenne Muscular Dystrophy
- TREAT-NMD Outcome Measures Meeting Newcastle 28th March 2008
- PPMD Annual Connect Conference_Philadelphia 12 - 14 July 2007
- EAMDA meeting 2007 - summary
- EAMDA meeting 2007 - full report
- TREAT-NMD Industrial Liaison Council meeting, Basel, October 07
- Skeletal Muscle MR Outcome Measures for Muscular Dystrophy Clinical Trials, September 07
- Report from the SMA Summit on Drug Development, September 07
- Report from the TREAT-NMD Governing Board meeting 2/3 July 2007
- TREAT-NMD workshop on outcome measures in experimental trials for DMD
- U.S. NIH workshop on translational research in muscular dystrophy
- 6th Monaco Round Table - clinical trials in DMD - full report
- 6th Monaco Round Table - clinical trials in DMD - summary by Kate Bushby
- First TREAT-NMD database curators meeting
- MD-NET Annual Meeting
- EuroBioBank/TREAT-NMD meeting report
- EURORDIS conference report
- The ethics of human clinical gene transfer: a CliniGene/CONSERT think tank
- Summary of the WP 8 meeting - production, toxicology, safety and delivery of therapeutics
- TREAT-NMD workshop on Spinal Muscular Atrophy - outcome measures, databases and standards of care
- Swedish network meeting for NMD
- Italian Telethon Convention 2007
- Brickless centre 2007 meeting
- TREAT-NMD clinical trials workshop June 08
- TREAT-NMD workshop report: preclinical testing for DMD
- EMEA SMA Outcome Measures Workshop 13.10.08
- Towards harmonisation of outcome measures for DMD and SMA within TREAT-NMD
- EAMDA meeting 2008
- TREAT-NMD DMD Workshop Report - Outcome Measures for Non Ambulatory DMD - 17th June 2009
- UK SMA Researchers Conference Report
Meetings & Events- CTCC Workshop Flyer 2010
- Myores Workshop Poster
- CTCC workshop flyer
- Provisional Programme
- Grant Application Form
- Grant Application Form
- Summer School Grant Application Form
- Course Registration
- Grant Application Form
- 6th Annual SMA Research Conference
- Conference programme flyer
- Registration Form
- Call for Posters
- Abstract Format Guidelines
- Update in Neuromuscular Disorders Flyer 2010
- Final conference programme
- Childhood Neuromuscular Disorders Barcelona 2010- Brochure
Neuromyology Curriculum- Minutes of the Task Force Neuromyology Curriculum Kick Off Meeting
Outcome measures- Article: Harmonisation of Outcome Measures for DMD and SMA
Patient Meetings- EAMDA AGM 2008
Project Ethics Council- PEC Terms of Reference
Rare NMD registries- 157th ENMC International Workshop on "Patient registries for rare inherited muscular disorders" (summary)
- Inventory of patient registries on rare inherited muscular disorders
ROM data collection- ROM User Account Request Form
SMA care- SMA précis - English
- SMA précis - German
- SMA précis - Dutch
- SMA précis - Russian
- SMA précis - Bulgarian
- SMA précis - Italian
- SMA précis - Polish
- SMA précis - Turkish
- Full consensus statement - English
- Family guide to the consensus statement
- SMA précis - Czech
SMA outcome measures EMEA workshop- TREAT-NMD/EMEA SMA Workshop Agenda
- EMEA SMA Outcome Measure Report 13.10.08
Social & Ethical Issues- From Empowerment for Patient Organisations to Empowered Patient Organisations
Stem Cell Tourism- Stem cell tourism - German Translation
Stem Cell Tourism - Hope versus Hype- Stem cell tourism - German Translation
The registries toolkit- A Handbook on SMA genetics
- UK Ethics Approval Application Form
- Instructions for Self-Report Form - DMD
- Self Report Form - DMD
- Patient Information on TREAT-NMD
- Self Report Form - SMA
- Consent Form
- Registry Protocol
- A Handbook on DMD genetics
- TREAT-NMD Registries Charter
- Mandatory Items for DMD and SMA
- Instructions for Self-Report Form - SMA
The TREAT-NMD global patient registries- Second annual curators’ training course Sept 2008
- German and UK TREAT-NMD Patient Registries for SMA Poster - 2009
- Third annual curator training course September 2009
- Patient registries and trial readiness in myotonic dystrophy - workshop report
Training and Education- Budapest training registration form
Training and Education Task Force- Minutes of the Task Force Neuromyology Curriculum Kick Off Meeting
- Minutes of the Task Force Neuromyology Curriculum Kick Off Meeting
