FDA grants accelerated approval to first drug for Duchenne muscular dystrophy: Exondys 51 (Eteplirsen)
Why and how does a corticosteroid, Emflaza (deflazacort), work in a supposedly degenerative condition like Duchenne Muscular Dystrophy?
Apparently, the dying muscle cells will activate cytotoxic T-cells, causing more accelerated inflammatory damage. For this reason, other immunosuppressants like Azathioprine and Cyclosporin are also occasionally used.
How is deflazacort different from other corticosteroids?
Apparently, it has less side effects on bone and fat metabolism.
But this study in mice examines 2 other chemicals: Osteoprotegerin immunoglobulin fragment complex (OPG-Fc), and a β2-agonist, Formoterol.