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

Kevin A StraussVincent J CarsonEmilienne BolettieriMariah EverettAshton BollingerLauren E BowserKeturah BeilerMillie YoungSimon EdvardsonNitay FraenkelAdele D'Amico Enrico Silvio BertiniLokesh LingappaDevyani ChowdhuryLinda P LowesMegan IammarinoLindsay N AlfanoKarlla W Brigatti

Published in: Annals of clinical and translational neurology (2023)

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.

Keyphrases

clinical trial
late onset
current status
big data
phase ii
artificial intelligence
early onset
deep learning
double blind