Audit of the TREAT-NMD global DMD and SMA registries: new insights into data collection methods


The global TREAT-NMD Alliance facilitates translational research and accelerates the development of therapies for patients with neuromuscular diseases (NMD). Key tools in this effort are the TREAT-NMD Duchene muscular dystrophy (DMD) and spinal muscular atrophy (SMA) global registries which collect data at multiple national sites, from over 40 countries across Europe, North America, Asia, Australasia and South America. Data are collected with clinical trial readiness in mind, information from genetically confirmed patients such as ambulation status; respiratory function and steroid use are collected.


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