Early data show follistatin gene transfer may be safe and effective in BMD and safe in IBM, in which efficacy has not yet been evaluated
Preliminary results from a trial to test the safety of injecting follistatin genes into the thigh muscles of adults with Becker muscular dystrophy (BMD)or sporadic (nongenetic) inclusion-body myositis (sIBM) suggest that the experimental therapeutic approach is safe in both types of patients
A follistatin gene therapy compound gains orphan drug status; potential improvements are reported for utrophin-based therapies, exon skipping and stem cell transplantation
Update (Jan. 23, 2013): The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.
Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.
Investigators in Columbus, Ohio, need to determine the best way to assess thigh-muscle strength in men with BMD before conducting a gene therapy study
A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants.
The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in...
Three sets of laboratory experiments investigating the effects of interfering with myostatin, a protein that limits muscle growth, have shown that this approach may have to be individualized with respect to different types and stages of muscular dystrophy, and that some myostatin suppression strategies may be better than others.