Potential Therapy for LGMD2B
A potential therapy for a specific type of muscular dystrophy (LGMD2B), caused by mutations in the DYSF gene leading to reduced dysferlin protein levels, involves membrane stabilization. Dysferlin is crucial for repairing the membrane of healthy muscle fibers, and its deficit results in chronic muscle inflammation.
In this study, the researchers compared the effects of two steroids, prednisolone and vamorolone (VBP15), on repairing dysferlin-deficient muscle fibers. Vamorolone, unlike prednisolone, successfully stabilized the muscle cell membrane, improved repair in dysferlin-deficient mice, and showed positive effects on muscle strength and late-stage myofiber loss.
On the other hand, prednisolone not only failed to stabilize the muscle cell membrane but also worsened muscle weakness and myofiber atrophy in mice. The findings suggest that vamorolone may be a more promising candidate for clinical trials in treating LGMD2B due to its superior efficacy and safety profile compared to prednisolone.
| Published | 9/9/2018 |
| Address | https://doi.org/10.1016/j.ymthe.2018.07.021 |
| Authors | Sen Chandra Sreetama,1 Goutam Chandra,1 Jack H. Van der Meulen,1 Mohammad Mahad Ahmad,1 Peter Suzuki,1 Shivaprasad Bhuvanendran,1 Kanneboyina Nagaraju,1,2 Eric P. Hoffman,1,2 and Jyoti K. Jaiswal1,3 |
