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

ABSTRACT Objective We created WiTNNess as a hybrid prospective/cross‐sectional observational study to simulate a clinical trial for infantile‐onset TNNT1 myopathy. Our aims were to identify populations for future trial enrollment, rehearse outcome assessments, specify endpoints, and refine trial log...

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Bibliographic Details

Published in:	Annals of Clinical and Translational Neurology
Main Authors:	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
Format:	Article
Language:	English
Published:	Wiley 2023-11-01
Online Access:	https://doi.org/10.1002/acn3.51884