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Patient network systems such as the US National Institutes of Healths Rare Diseases Clinical Research Network 42 and the National Rare Diseases Registry System of China 43 facilitate access to geographically dispersed populations

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These murine models were initially presented with corticosteroids, which reportedly contributed to severe muscular damage in these murine models

Additional Reviews Already Scheduled The announcement also outlines next steps, making clear that this is not the final word on these therapies