Sarepta Therapeutics' next Duchenne muscular dystrophy (DMD) med seems to be doing the job of spurring dystrophin expression. But do any of the data for SRP-5051 matter given the recent approval of ...
Since utrophin compensates for lack of dystrophin in mdx mice, which results in a milder phenotype of muscular dystrophy compared to humans, the mdx/utrn-/- mouse has been developed to mimic early ...