Session 17 kate_bushby

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  • The draft version of the call was published in LSH programme. Specific topic. It already outlined the major objectives for the proposal and it defined the specific type of project also known as the instrument for the proposal – a NoE. Topic, text – this is almost a definition of translational research in neuromuscular diseases. And tools are defined. When published - There were two questions: what is a NoE and are we as the NM community going to apply for a NM NoE?
  • The map shows the 11 partner countries, From 21 to 22 partners, 164 deliverables, 29 workshops organized by the partners – not about partners, who have to do the work, but a project for all those interested in NMD and the development and realization of clinical trials – still a difficult concept, academics used to work together when there was money or there were authorships to share...from CoI to members and a membership charter
  • TREAT-NMD is actively reaching out to contact more peolpe in the NMD field - newsletter
  • Everything of relevance and even many things of irrelevance in the application process of a grant to the EU is well defined. In summary a NoE. This means: aims to bring together those partners in the field that are known to be excellent, integrate them with the aim to form a lasting or permanent infrastructure that helps to maintain and improve excellence in the specific field and a result of this specific NoE should be the elaboration of new therapies for NMD
  • Everything of relevance and even many things of irrelevance in the application process of a grant to the EU is well defined. In summary a NoE. This means: aims to bring together those partners in the field that are known to be excellent, integrate them with the aim to form a lasting or permanent infrastructure that helps to maintain and improve excellence in the specific field and a result of this specific NoE should be the elaboration of new therapies for NMD
  • Concurrent to developing the network tools to ensure trial readiness we are collaborating with industry in a numbers areas. Their interest is broad: design of clinical trials, access to well characterised and standardised patient groups, pre-clinical testing, and production of therapeutic agents. Current collaborations include: PTC Therapeutics- PTC124 Trophos Acceleron Pharma Prosensa AVI BioPharm Santhera Summit Genethon Genosafe To find out more about how we work with industry please visit our Industry pages (launched next week)
  • Session 17 kate_bushby

    1. 1. TREAT-NMD Translational Research in Europe – Assessment and Treatment of Neuromuscular Diseases advancing diagnosis, care and treatment for people with neuromuscular diseases around the world A network of excellence to catalyse research infrastructure globally
    2. 2. Rare neuromuscular diseases <ul><li>Individually rare </li></ul><ul><li>Frequently chronically disabling </li></ul><ul><li>Few therapies based on best levels of evidence </li></ul><ul><li>Strong patient advocacy voice </li></ul><ul><li>Charitable funding of research </li></ul><ul><li>Strong pipeline of research projects leading to the promise of translational research </li></ul>
    3. 3. The Life Sciences, Genomics And Biotechnology For Health: 4th call <ul><li>A Network of Excellence is an instrument for strengthening excellence by tackling the fragmentation of European research. </li></ul><ul><li>This network of excellence will aim at sharing expertise between basic and clinical academics and industrial partners in order to develop technological and methodological tools with a view to accelerate the elaboration of new therapies for rare neuromuscular diseases… </li></ul>LSH-2005-2.1.1-7: Rare inherited neuromuscular diseases: FROM MOLECULAR BASIS TO CUTTING EDGE THERAPIES
    4. 4. TREAT-NMD Partners
    5. 5. TREAT-NMD members ………… Parent Project Association , Association Muscular Dystrophy, CINRG , SMA Trust, University Hospital Basel , University of Utah, Washington University in St Louis , UPA! Cura Duchenne, University of Bucharest , Virgen Del Rocio University Hospital, University of Ulm , Jennifer Trust for SMA, Muscular Dystrophy Campaign , Asociace Muskularnich Dystrofiku v CR, Medical University of Warsaw , University Children's Hospital, SMA Trust , Kolpingova rodina Smecno, SMA Deutschland , Myotonic Dystrophy Foundation, Wake Forest University , Australian Neuromuscular Research Institute, Children With SMA , Cure CMD, Centre Hospitalier Regional de la Reunion , Clinic for Child Neurology, Martin House Hospice , DuchenneConnect, University of Glasgow , Hospital Pediatria J.P. Garrahan……………
    6. 6. >3200 recipients, >1000 pdf downloads per month Outreach: newsletter and website
    7. 7. Network tools and resources are generated by expert consensus within the community for use by the community and interested parties for the benefit of patients <ul><li>Focus on rare inherited neuromuscular diseases- addressing the </li></ul><ul><li>shared challenges facing the field </li></ul><ul><li>Involvement of patient/ advocacy groups as partners in activities </li></ul><ul><li>Co-working with industry </li></ul><ul><li>A platform for further academic initiatives </li></ul><ul><li>Close interactions with the regulatory authorities </li></ul>
    8. 8. Network tools are resources aim to accelerate therapy delivery for NMD
    9. 9. Tools and resources add value across the field
    10. 10. <ul><li>Current collaborations include: </li></ul><ul><li>Acceleron </li></ul><ul><li>AVI Biopharma </li></ul><ul><li>Biomarin </li></ul><ul><li>Debiopharm </li></ul><ul><li>Genzyme </li></ul><ul><li>Prosensa/ GSK </li></ul><ul><li>PTC Therapeutics </li></ul><ul><li>Santhera </li></ul><ul><li>Trophos…….. </li></ul>Industry involvement Current activities include: Preclinical and biochemical OM consensus Patient and trial site enquiries Patient recruitment SAB meetings OM selection and training CRO support Preparing funding applications TACT appraisal
    11. 11. International academic collaborations <ul><li>EUROBIOBANK (Access to patient samples) </li></ul><ul><li>NMD-Chip (Diagnostics) </li></ul><ul><li>BIO-NMD (Biomarker identification) </li></ul><ul><li>CARE-NMD (Implementation of standards of care) </li></ul><ul><li>FOR-DMD (NIH steroid trial in DMD) </li></ul><ul><li>Jain Foundation Dysferlinopathy natural history project </li></ul>
    12. 12. Observations on the utility of the network of excellence tool <ul><li>Funding structure allows partners to work on infrastructure development and implementation </li></ul><ul><li>Model is appreciated and indeed envied in other parts of the world </li></ul><ul><ul><li>This facilitates collaboration with other parties </li></ul></ul><ul><li>Lack of emphasis on research means that distinction from other funding structures can be clear </li></ul><ul><li>EU expects a “durable infrastructure” </li></ul><ul><ul><li>Metrics for successful networks and means to sustain them need to be set in place as high priority to protect investment </li></ul></ul>
    13. 13. TREAT-NMD global partners

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