Motor improvements in SMA adults treated with Spinraza paralleled a boost in electrical impulses along nerves that supply ...

A majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe muscle degeneration ... for spinal muscular atrophy (SMA) to the list ...

EPI-321, the startup’s lead program, uses CRISPR tools to stop errant expression of a gene implicated in the muscle-wasting condition. Epicrispr will start a Phase 1 trial this year in New Zealand, ...