Sarepta Therapeutics has announced preliminary results from the MYO-101 trial. Three children with LGMD type 2E have been dosed with the gene therapy so far. Safety concerns related to the liver were reported in two participants, but these were quickly resolved with steroid treatment. Muscle biopsies taken after two months showed high levels of beta-sarcoglycan, the protein that is missing in LGMD type 2E. Creatine kinase levels also decreased, suggesting a reduction in muscle damage. Although these results are promising, it’s still early days and we don’t know what effect the gene therapy has on muscle function.
For more information, read Sarepta’s press release.