Results 221 to 230 of about 321,114 (362)

Misfolding of fukutin-related protein (FKRP) variants in congenital and limb girdle muscular dystrophies. [PDF]

open access: yesFront Mol Biosci, 2023
Esapa CT   +13 more
europepmc   +1 more source

The genetic basis of undiagnosed muscular dystrophies and myopathies: Results from 504 patients

open access: yesNeurology, 2016
M. Savarese   +57 more
semanticscholar   +1 more source

A model‐informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy

open access: yesCPT: Pharmacometrics &Systems Pharmacology, EarlyView.
Abstract Quantitative model‐based clinical trial simulation tools play a critical role in informing study designs through simulation before actual execution. These tools help drug developers explore various trial scenarios in silico to select a clinical trial design to detect therapeutic effects more efficiently, therefore reducing time, expense, and ...
Jongjin Kim   +10 more
wiley   +1 more source

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