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In 2007 TREAT-NMD (Translational Research Europe-Assessment and Treatment of neuromuscular diseases) was set up as an EU network of excellence to bring more harmonisation and integration of efforts towards therapy development for rare inherited neuromuscular diseases. The network now encompasses collaborations worldwide and reaches out to an audience of over 2,500 via a regular newsletter. The network activities address the need to enhance the infrastructure for running trials and delivering treatments in these rare disorders. Consensus guidelines on the use of animal models and high throughput screening to direct therapy development have improved our understanding of the preclinical environment. At the clinical level, a major effort has gone into the harmonisation of existing and new databases and registries for patients with neuromuscular disorders. Over 20 national registries are collaborating on global databases for Duchenne muscular dystrophy and spinal muscular atrophy and more are in development. The “toolkit” for registry development is now being applied to other diseases where clinical trials may be on the horizon. Similarly, standards of care recommendations started with an emphasis on DMD and SMA but now are being extended to other disease groups. Other activities include the evaluation of clinical outcome measures and a registry of sites collaborating on care and delivery of trials. Interest is high from industry and academic groups keen to run trials.
We believe that TREAT-NMD is developing into an important worldwide platform for the delivery of therapies in these diseases, and are always ready to welcome new members and develop new ideas for the integration of different diseases or approaches to therapy.