Abstract:The previous TREAT-NMD Duchenne muscular dystrophy (DMD) dataset was designed mainly to support clinical trial feasibility and recruitment. Therapeutic developments, anticipation of post-marketing surveillance and interest in better understanding the disease natural history, meant the dataset needed to be updated to meet these new requirements. To reach consensus on a new version of data items that can be shared amongst the stakeholder community, and used by all registries collecting data from DMD patients, a … Show more
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