Randomized phase 2 trial· 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
Statland JM|Campbell C|Desai U|Karam C|Díaz-Manera J|Guptill JT|Korngut L|Genge A|Tawil RN|Elman L|Joyce NC|Wagner KR|Manousakis G|Amato AA|Butterfield RJ|Shieh PB|Wicklund M|Gamez J|Bodkin C|Pestronk A|Weihl CC|Vilchez-Padilla JJ|Johnson NE|Mathews KD|Miller B|Leneus A|Fowler M|van de Rijn M|Attie KM
Muscle & Nerve
- PubMed
- 35428982
- Evidence level
- moderate
- Verification
- verified
Summary
A randomized phase 2 study evaluated locally injected ACE-083 in adults with facioscapulohumeral muscular dystrophy.
Limitations
Disease-specific population|Local intramuscular administration|No consistent functional benefit|Development programme discontinued