This article explores DYSF mutations and Miyoshi Myopathy, detailing the role of dysferlin in muscle membrane repair and how its deficiency leads to muscle degeneration.
This article discusses LGMD2I and LGMD2B, two subtypes of limb-girdle muscular dystrophy, their genetic causes, symptoms, and current management options.
This article discusses multiple muscle diseases, including ZNF9, BMD, MC-AD, DMD, and TMD, highlighting their genetic causes, symptoms, and disease progression.
This article explores various muscle diseases, including DM1, DM2, and DMx, detailing their genetic causes, symptoms, and the role of DMPK in myotonic dystrophy.
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.
This study explores the potential of membrane stabilization therapy for LGMD2B using vamorolone, showing it outperforms prednisolone in muscle repair and strength restoration.