The key difference between BMD and DMD is that in DMD there is no functional dystrophin at all ... inhibiting fast myofiber (type II) myosin." This makes it genotype-agnostic, and also gives ...

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to ... caused by the absence or loss of functional dystrophin. By minimizing the progressive muscle ...