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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
Rare neuromuscular diseases ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
The Life Sciences, Genomics And Biotechnology For Health: 4th call ,[object Object],[object Object],LSH-2005-2.1.1-7: Rare inherited neuromuscular diseases:   FROM MOLECULAR BASIS TO CUTTING EDGE THERAPIES
TREAT-NMD Partners
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……………
>3200 recipients, >1000 pdf downloads per month Outreach: newsletter and website
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 ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Network tools are resources aim to accelerate therapy delivery for NMD
Tools and resources add value across the field
[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],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
International academic collaborations ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Observations on the utility of the network of excellence tool ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
TREAT-NMD global partners

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Session 17 kate_bushby

  • 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.
  • 3.
  • 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. >3200 recipients, >1000 pdf downloads per month Outreach: newsletter and website
  • 7.
  • 8. Network tools are resources aim to accelerate therapy delivery for NMD
  • 9. Tools and resources add value across the field
  • 10.
  • 11.
  • 12.

Notas del editor

  1. 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?
  2. 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
  3. TREAT-NMD is actively reaching out to contact more peolpe in the NMD field - newsletter
  4. 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
  5. 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
  6. 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)