Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease.
Florian Patrick ThomasThomas H BrannaganRussell J ButterfieldUrvi DesaiAli A HabibDavid N HerrmannKaty J EichingerNicholas E Johnson-ClChafic KaramAlan PestronkColin QuinnMichael E ShyJeffrey M StatlandSub H SubramonyDavid WalkKatherine Stevens-FavoriteBarry MillerAshley LeneusMarcie FowlerMarc van de RijnKenneth M AttiePublished in: Neurology (2022)
This study provides Class II evidence that intramuscular ACE-083 is safe, well tolerated, and increases total muscle volume after 6 months of treatment in adults with CMT1 or CMTX.