International Study of TNNT1 Myopathy Establishes Operational Framework for Future Clinical Trials

STRASBURG, PA – A new study summarizes WiTNNess – a hybrid prospective/cross-sectional observational study of TNNT1 myopathy, a rare and lethal neuromuscular disease that causes progressive muscle weakness and stiffness, while cognitive and social development remain normal. The study aimed to identify clinically meaningful endpoints for future therapeutic trials following recent guidelines on natural history studies to support clinical trial readiness. The study was a broad collaborative effort led by clinicians and researchers at the Clinic for Special Children that included participants from around the globe and appears in the current issue of Annals of Clinical and Translational Neurology.

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