Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

@ATSPeds Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistro… https://t.co/t4muJhWjP2

@RNAiAnalyst Is this (n=1) at all comparable to Sarepta’s dystrophin expression level? From a very brief google I don’t see raw expression data for Sarepta but the standard deviations suggest this is essentially useless as a quantitative endpoint? https://

Gene transfer therapy helps introduce new genetic material into cells to recover the function of a certain cell. Dr. Perry Shieh is involved in a study that dives into how this form of therapy can be applied to muscular dystrophy. #NeuroTwitter https://t

New #FronteirsinCellDevBiology paper: Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy. https://t.co/WFDZN8MnII