Disease Progression in Charcot-Marie-Tooth Disease Related to MPZ Mutations: A Longitudinal Study.

Vera FridmanStefan SillauJacob BockhorstKaitlin SmithIsabella MoroniEmanuela PaglianoChiara PisciottaGuiseppe PiscosquitoMatilde LauráFrancesco MuntoniChelsea BaconShawna FeelyTiffany GriderLaurie GutmannRosemary ShyJanel WilcoxDavid N HerrmannJun LiSindhu RamchandrenCharlotte J SumnerThomas E LloydJohn DayCarly E SiskindSabrina W YumReza SadjadiRichard S FinkelSteven S SchererDavide PareysonMary M ReillyMichael E Shynull null

Published in: Annals of neurology (2022)

CMTES is sensitive to change over time in adult patients with axonal but not demyelinating forms of MPZ neuropathy. Change in CMTES was greatest in patients with moderate baseline disease severity. These findings will inform future clinical trials of MPZ neuropathies. ANN NEUROL 2022.

Keyphrases

clinical trial
spinal cord injury
current status
high intensity
neural network
open label