Few patients with a neuromuscular condition currently benefit from effective or curative therapies, and many do not have a definite diagnosis. Uniting healthcare and research would begin to address this and is therefore in the best interests of patients.
EURO-NMD members are not only specialist healthcare providers but also among the world’s leading researchers in the neuromuscular field and as such recognise the value in a network like EURO-NMD. Our healthcare providers share a wealth of experience in participating in collaborative networks and are well positioned to identify knowledge gaps and prioritise research to address them. Research in the neuromuscular field must be collaborative because of the rarity of individual conditions, patients and resources, and we will build upon valuable work already undertaken by making use of established transnational infrastructures and research networks.
Translational research will bring outcomes of basic research into the clinic through the many research-active expert centres in EURO-NMD, which will act as a “research-ready” network of expertise holding cohorts of patients with standardized clinical data, standardized baseline care and available biosamples. For our research to be successful and clinically meaningful, it requires the active participation of patients and health professionals alike.
We aim to keep our patients informed about research both general and specific to their condition. We will also highlight opportunities they will have to participate in this research which will could be in the form of biobanks, registries, natural history studies, clinical trials.
Networks of note
EuroBioBank network, the first operating network of biobanks in Europe providing human DNA, cell and tissue samples as a service to the scientific community conducting research on rare diseases. It is the only network dedicated to rare disease research in Europe. About 130,000 samples are available across the network and can be requested via the online catalogue. Approximately 13,000 samples are collected each year and 7,000 samples distributed in Europe and beyond. The biological samples are obtained from patients affected by rare diseases, including rare neuromuscular disorders.
RD-Connect is a unique global infrastructure project that links up databases, registries, biobanks and clinical bioinformatics data used in rare disease research into a central resource for researchers worldwide. In a six-year project funded by the European Union but uniting researchers across the world, it will develop an integrated research platform in which complete clinical profiles are combined with -omics data and sample availability for rare disease research, in particular research funded under the International Rare Diseases Research Consortium (IRDiRC).
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. Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.