Miyoshi myopathy (MM) is an adult onset, recessive inherited distal muscular dystrophy that we have mapped to human chromosome 2p13. We recently constructed a 3-Mb P1-derived artificial chromosome ...

Edgewise Therapeutics’ lead candidate has been shown to reduce levels of a biomarker related to skeletal muscle damage in a phase 2 trial. The biotech had been testing sevasemten, an orally ...