WiTNNess: An international natural history study of infantile-onset TNNT1 myopathy.

Kevin A Strauss,Vincent J Carson, Emilienne Bolettieri,Mariah Everett, Ashton Bollinger,Lauren E Bowser,Keturah Beiler,Millie Young,Simon Edvardson, Nitay Fraenkel,Adele D'Amico, Enrico Bertini,Lokesh Lingappa,Devyani Chowdhury,Linda P Lowes,Megan Iammarino,Lindsay N Alfano,Karlla W Brigatti

Annals of clinical and translational neurology（2023）

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Abstract

WiTNNess allowed us to streamline and economize the collection of historical control data without compromising scientific rigor, and thereby establish a sound operational framework for future clinical trials.

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