This study highlights vamorolone’s ability to improve Becker muscular dystrophy by reducing inflammation and increasing dystrophin levels in BMX model mice.
This study introduces the BMX mouse model for Becker muscular dystrophy, providing a new tool for studying disease mechanisms and testing potential therapies.
This study examines dystrophin and dystroglycan protein turnover after exon skipping therapy in DMD, highlighting protein stability changes and implications for gene correction strategies.