Donate to the TREAT-NMD Alliance

  • Annemieke Aartsma-Rus
    Annemieke Aartsma-Rus
    Dr. Annemieke Aartsma-Rus is associate professor at the Department of Human Genetics and leader of...
  • Kevin Flanigan
    Kevin Flanigan
    Dr. Kevin Flanigan is a Professor of Neurology and Pediatrics at the Ohio State University,...

TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Advances in research and industry are moving quickly and TREAT-NMD hopes to have the infrastructure in place to ensure that these developments have the greatest possible impact on the patients that need them. The network, launched in 2007, has focused on creating and implementing the tools that industry, clinicians and scientists can use to bring novel therapeutic approaches through preclinical development and into the clinic. To support this TREAT-NMD is working on establishing the best possible standards of care for neuromuscular patients worldwide.

“Your donation will work towards clinical application therapies for neuromuscular disorders, such as the exon skipping approach for DMD I am involved in. For this a global effort is needed to maintain and improve on patient registries, obtain useful and meaningful outcome measures for ambulant and non-ambulant patients, as well as coordination on preclinical and clinical tests to ensure a concerted effort rather than unnecessary duplication of work.” (Annemieke- Chair of the TREAT-NMD Alliance)

Help us to continue working towards therapies for neuromuscular disorders - donate here

The TREAT-NMD Alliance was formed in 2012 following on from an initial period of funding from the European Commission. The new Alliance was intially run by an Executive Committee chaired by Professor Hanns Lochmϋller with Annemieke Aartsma-Rus as vice chair. The network has many European and global partners and is always keen to work with new collaborators.

The TREAT-NMD Alliance has published a three year action plan with clearly defined goals and objectives in order to ensure the network moves forward with its aim for global ‘trial readiness’ in neuromuscular disease. The action plan can be broken down into a number of key areas and you can click on the diagram below to learn more.

Three year plan BioBank Outcome Measures Patient Registries TACT Joint Research Website and Communications Standard Operating Procedures

Recent years have seen rapid developments in the neuromuscular field and a corresponding surge in interest from the pharmaceutical industry. Promising preclinical results also raise the potential for new therapies in the near future. The TREAT-NMD Alliance is working towards a global goal of trial readiness, and in doing this faces barriers from the different sectors involved. For patients, promising research results have still not been translated into the treatments they hope for, while a lack of standardised care guidelines prevents many from receiving optimal care.

“By acting as catalyst in developing standards of care, advancing clinical research, providing expertise in outcome measure development and leading the creation of national patient registries, TREAT-NMD is a key partner in the development of ataluren and a guide to PTC as we advance our efforts in Duchenne Muscular Dystrophy.” (PTC Therapeutics)

For the biomedical industry, identifying the investigators and sites with the relevant expertise and accessing the appropriate patient cohorts for clinical trials has been a significant challenge.

For clinicians and researchers, lack of support tools such as validated clinical outcome measures or standard operating procedures for research protocols has held back therapeutic development.

The TREAT-NMD Alliance addresses all these issues, and works towards the best possible outcome while aiming to provide industry and researchers with the tools they need. In doing this we unite the stakeholders across the community and provide an infrastructure that is accelerating research and therapy development, increasing collaboration, improving patient care and helping to support ‘clinical trial readiness’ on an international scale. The TREAT-NMD Alliance has developed from its initial European roots to become a truly global platform bringing together leading specialists, patient groups and industry representatives to ensure preparedness for the trials and therapies of the future while promoting best practice today. All funds that we receive will help the TREAT-NMD Alliance not only achieve their three year action plan but also help the Alliance to sustain future developments; improving the standards of care and treatment for people with neuromuscular diseases globally.

“Parent Project onlus is a patients’ organization based in Italy focused on DMD and BMD. TREAT NMD Alliance has been crucial for development and promotion of standards of care, highlighting the importance of collaboration between patient organizations, researchers, clinicians and companies. Above all, it has been a catalyzer for the creation of National Registries and for their global harmonization, which has been essential for the development of clinical trials. For all these reasons it is important to support TREAT NMD Alliance’s work, because there is still much to be done.”  (Parent project Onlus.)"

If you would like to discuss making a donation to the TREAT-NMD Alliance in more detail please contact Stephen Lynn.

If you would like to make a donation today please click here.

The secretariat for the TREAT-NMD Alliance is based at Newcastle University. You can find out more about Newcastle University's involvement in Neuromuscular Research here.

 
12 Apr 2017