TREAT-NMD

TREAT-NMD
Translational Research in Europe - Assessment and Treatment of Neuromuscular diseases
Keywords	Neuromuscular diseases, clinical research
Project type	Network of Excellence
Funding agency	European Commission
Reference	FP6-036825
Objective	Improving trial-readiness worldwide, advancing patient diagnosis and care and accelerating pre-clinical research
Project coordinator	Institute of Genetic Medicine, Newcastle University
Participants	Universities, pharmaceutical companies, patient organisations
Website	www.treat-nmd.org

TREAT-NMD (Translational Research in Europe, Assessment and Treatment for NeuroMuscular Disorders) is a global academic network that focuses on advancing research in neuromuscular disorders.[1] It was established in 2007 with its coordination centre at the Newcastle University.[2] As of 2018, the network comprises over a hundred research centres and patient organisations from 54 countries[3] as well as independent academics and patient representatives.[4] The network's aim is to provide infrastructure to accelerating research through supporting collaboration between its members.[5] Its main goals include improving trial-readiness worldwide, advancing patient diagnosis and care and accelerating pre-clinical research.[6][7]

Through patient registries, TREAT-NMD provides genotype–phenotype correlation between genetic mutations and neuromuscular disease burden.[8]

References

1. Leary, Rebecca; Oyewole, Anne; Bushby, Katharine; Aartsma-Rus, Annemieke (6 July 2017). "Translational Research in Europe for the Assessment and Treatment for Neuromuscular Disorders (TREAT-NMD)". Neuropediatrics. 48 (4): 211–220. doi:10.1055/s-0037-1604110. PMID 28683502. S2CID 19283049.
2. Bushby, Katharine; Lynn, Stephen; Straub, Volker (July 2009). "Collaborating to bring new therapies to the patient--the TREAT-NMD model". Acta Myologica. 28 (1): 12–5. PMC 2859629. PMID 19772190.
3. "TREAT-NMD Alliance Members – Organizations". TREAT Neuromuscular Network. Retrieved 13 June 2018.
4. "TREAT-NMD Alliance Members – Individuals". TREAT Neuromuscular Network. Retrieved 13 June 2018.
5. "TREAT-NMD : About the TREAT-NMD network". TREAT Neuromuscular Network.
6. Willmann, Raffaella; Rüegg, Markus A.; Fairclough, Rebecca J.; Davies, Kay E; Possekel, Stefanie; Meier, Thomas (October 2008). "T.P.3.03 TREAT-NMD-Activity 7: Accelerate preclinical phase of new therapeutic treatment development". Neuromuscular Disorders (Conference abstract). 18 (9–10): 794. doi:10.1016/j.nmd.2008.06.242. S2CID 54330245.
7. Mercuri, Eugenio Maria; Mayhew, Anna G; Muntoni, Francesco; Straub, Volker; Van Ommen, Gert-Jan; Voit, Thomas; Bertini, Enrico; Bushby, Katherine (November 2008). "Towards harmonisation of outcome measures for DMD and SMA within TREAT-NMD; report of three expert workshops: TREAT-NMD/ENMC workshop on outcome measures, 12th--13th May 2007, Naarden, The Netherlands; TREAT-NMD workshop on outcome measures in experimental trials for DMD, 30th June--1st July 2007, Naarden, The Netherlands; conjoint Institute of Myology TREAT-NMD meeting on physical activity monitoring in neuromuscular disorders, 11th July 2007, Paris, France". Neuromuscular Disorders (Workshop report). 18 (11): 894–903. doi:10.1016/j.nmd.2008.07.003. PMID 18818076.
8. Thompson R, Robertson A, Lochmüller H (2017). "Natural History, Trial Readiness and Gene Discovery: Advances in Patient Registries for Neuromuscular Disease". Rare Diseases Epidemiology: Update and Overview. Advances in Experimental Medicine and Biology. Vol. 1031. pp. 97–124. doi:10.1007/978-3-319-67144-4_5. ISBN 978-3-319-67142-0. PMID 29214567.

Further reading

• Heslop, Emma; Csimma, Cristina; Straub, Volker; McCall, John; Nagaraju, Kanneboyina; Wagner, Kathryn R.; Caizergues, Didier; Korinthenberg, Rudolf; Flanigan, Kevin M. (23 April 2015). "The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development". Orphanet Journal of Rare Diseases. 10 (1): 49. doi:10.1186/s13023-015-0258-1. PMC 4417237. PMID 25902795.
• Bladen, Catherine L.; Rafferty, Karen; Straub, Volker; Monges, Soledad; Moresco, Angélica; Dawkins, Hugh; Roy, Anna; Chamova, Teodora; Guergueltcheva, Velina (26 August 2013). "The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia". Human Mutation. 34 (11): 1449–1457. doi:10.1002/humu.22390. PMID 23913485. S2CID 38772058.
• Aartsma-Rus, Annemieke; Mercuri, Eugenio; Vroom, Elizabeth; Balabanov, Pavel (April 2018). "Meeting report of the "Regulatory Exchange Matters" session at the 5th International TREAT-NMD Conference:: Lessons in communication: How an early dialogue between patients, regulators and academics can further therapy development for neuromuscular disorders Freiburg, Germany, 27-29 November 2017". Neuromuscular Disorders. 28 (7): 619–623. doi:10.1016/j.nmd.2018.04.009. PMID 29778308.