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, as well as on establishing best-practice care for neuromuscular patients worldwide.
EURORDIS is a non-governmental patient-driven alliance of patient organisations representing 687 rare disease patient organisations in 63 countries. They are the voice of 30 million people affected by rare diseases throughout Europe.
European Medicines Agency (EMA)
The European Medicines Agency (EMA) is a decentralised agency of the European Union, located in London. The Agency is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union. It began operating in 1995.
EMA’s main responsibility is the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use.
The European Neuromuscular Centre (ENMC) was founded in 1992 by a group of European patient associations that dedicated itself to bring leading researchers and clinicians from all over the world together. The ENMC is unique within the scientific world to achieve this by financing and organising workshops. Topics of the workshops vary from outcome measures, clinical trial readiness and preclinical studies, to diagnosis and care for all neuromuscular diseases (e.g. spinal muscular atrophy, Duchenne muscular dystrophy, limb girdle dystrophies and congenital muscular dystrophies).
EURO-NMD is a European Reference Network for the thematic grouping of rare neuromuscular diseases (NMDs). It unites 61 of Europe’s leading NMD clinical and research centres in 14 Member States and includes highly active patient organisations.
More than 100,000 NMD patients are seen annually by the ERN. The network addresses harmonising and implementing standards for clinical and diagnostic best practice, improving equity of care provision across Member States, decreasing time to diagnosis, increasing cost efficiency through better care pathways, access to specialist training and education, application of eHealth services, development and application of care guidelines, facilitating translational and clinical research, harmonising data and samples for research reuse, and sharing of high-quality data.