Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.
Jeffrey M StatlandCraig CampbellUrvi DesaiChafic KaramJordi Díaz-ManeraJeffrey T GuptillLawrence KorngutAngela GengeRabi N TawilLauren ElmanNanette C JoyceKathryn R WagnerGeorgios E ManousakisAnthony A AmatoRussell J ButterfieldPerry B ShiehMatthew WicklundJosep GamezCynthia BodkinAlan PestronkConrad C WeihlJuan J Vilchez-PadillaNicholas E JohnsonKatherine D MathewsBarry MillerAshley LeneusMarcie FowlerMarc van de RijnKenneth M AttiePublished in: Muscle & nerve (2022)
Significant increases in TMV with ACE-083 vs placebo did not result in consistent functional or PRO improvements with up to 12 months of treatment.