Open AccessRandomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Author(s) -
Florian P. Thomas,
Thomas Brannagan,
Russell J. Butterfield,
Urvi Desai,
Thomas H. Brannagan,
David N. Herrmann,
Katy Eichinger,
Nicholas Johnson,
Chafic Karam,
Alan Pestronk,
Colin Quinn,
Michael E. Shy,
Jeffrey Statland,
S. H. Subramony,
David Walk,
Katherine Stevens-Favorite,
Barry Miller,
Ashley Leneus,
Marcie Fowler,
Marc van de Rijn,
Kenneth M. Attie
Publication year - 2022
Publication title -
neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.91
H-Index - 364
eISSN - 1526-632X
pISSN - 0028-3878
DOI - 10.1212/wnl.0000000000200325
Subject(s) - placebo , medicine , clinical endpoint , tolerability , adverse effect , randomized controlled trial , pathology , alternative medicine
To determine whether locally acting ACE-083 is safe, well tolerated, and increases muscle volume, motor function, and quality of life (QoL) in adults with Charcot-Marie-Tooth disease (CMT) type 1.